Premio Europeo di Genetica Umana 2025 – XIV Edizione – Prof.ssa Marina Cavazzana

Marina CAVAZZANA

Born 01/01/1959, Venice, Italy Nationality French [email protected] +33 (0)1 42 75 42 52

Professional addresses

Institute Imagine – U1163 INSERM 24, boulevard du Montparnasse 75015 Pari, France

Hôpital Necker-Enfants Malades Département de Biothérapie 149, rue de Sèvres 75015 Paris, France

Marina CAVAZZANA, main research and clinical interests are the development of the hematopoietic immune system, and cell and gene therapy for inherited and acquired disease of the hematopoietic system. Her group studies the means to improve the clinical results of hematopoietic stem cell transplantation, crossing HLA-barriers, and the differentiation of mouse and human stem cells towards lymphocyte lineages. She has initiated several clinical trials based on the use of ex vivo gene modified hematopoietic stem cells to treat patients with inherited disorders, the preliminary clinical results of which are encouraging. Her work was rewarded by the American Society of Hematology (Award on Clinical Research in Gene Therapy in 1999), by the French Academy of Sciences (Special Medical Award in 2000 and Jean-Pierre Lecocq Award on Gene Therapy in 2004). She was awarded the title of Officier de l’Ordre National de la Légion d’honneur in 2011, and given the Irène Joliot Curie 2012 award “Scientific Women of the Year” (Science Academy and French Ministry of Education and Research). She was also awarded with the French National Academy of Medicine in 2016 and the 2017 Ernest Beutler Lecture and Prize for Clinical Science (American Society of Hematology). She has also been elected as an International member of the National Academy of Medicine in 2019, and member of the French Academy of Medecin in 2023. She is the co-funder and strategic clinical development advisor of SmartImmune, a clinical-stage biotechnology company developing ProTcell, a thymus-empowered cell therapy platform to rapidly re-arm the immune system, enabling next-generation allogeneic T-cell therapies to help patients with life-threatening diseases such as high-risk blood cancers and primary immunodeficiencies.

ACADEMIC APPOINTMENTS

2014-2019 Director of the Research of Human Lymphohematopoiesis Laboratory INSERM U1163 at Imagine Institute (Co-head Dr Isabelle ANDRE)
2006-Present Coordinator of the Biotherapy Clinical Investigation Center (CIC), GHU (APHP-INSERM)
2003-Present Director of the Department of Biotherapy, Necker-Enfants Malades Hospital (Paris)
1994-2014 Director of the Research Team No.6 (new approach to primary immunodeficiency), INSERM Unit 768

TRAINING

Medical training

2001 Qualification in Transfusion Technologies, Franche-Comté University
2000 Professor of Haematology/ Innovative Therapies – Full Practitionner, Paris Descartes University (now Paris Cité University, France)
1985-1987 Clinical Oncology Certification, Paris VII University (now Paris Cité University)
1983-1987 Residency in Paediatric, University of Padua, Italy
1983 Nationnal Board, Pediatrics (MD)

Research Training

1997 Ability to Lead a Research – Paris Descartes University (now Paris Cité University)
1989-1993 PhD, Paris VII University (now Paris Cité University, France)
1988 Immunobiotechnologies, Immunogenetics & Blood Transfusion- DEA
1987 Master in Human Biology / Experimental Oncology, Paris XI University
1984 Master in Human Biology / Haematology, Paris VII University (Paris Cité University)

EXPERTISE

Membership in the Scientific Societies and Scientific Responsabilities:

5 MAJOR PUBLICATIONS in gene therapy field in the last 5 years

Sobrino S.^*, Magnani A^,*,Semeraro M, Martignetti L , Cortal A, Denis A , Couzin C, Picard C, Bustamante J, Magrin E, Joseph L, Roudaut C, Gabrion A, Soheili T, Cordier C, Lortholary O, Lefrere F, Rieux-Laucat F, Casanova JL, Bodard S, Boddaert N, Thrasher AJ, Touzot F, Taque S, Suarez F, Marcais A, Guilloux A, Lagresle-Peyrou C, Galy A ^*, Rausell A^,**, Blanche S ^,*, Cavazzana M^*,#, Six E^,*Severe hematopoietic stem cell inflammation compromises chronic granulomatous disease gene therapy Cell Reports Medicine 2023 (*These authors contributed equally ^#Corresponding author)
Magrin E, Semeraro M, Hebert N, Joseph L, Magnani A, Chalumeau A, Gabrion A, Roudaut C, Marouene J, Lefrere F, Diana JS, Denis A, Neven B, Funck-Brentano I, Negre O, Renolleau S, Brousse V, Kiger L, Touzot F, Poirot C, Bourget P, El Nemer W, Blanche S, Tréluyer JM, Asmal M, Walls C, Beuzard Y, Schmidt M, Hacein-Bey-Abina S, Asnafi V, Guichard I, Poirée M, Monpoux F, Touraine P, Brouzes C, de Montalembert M, Payen E, Six E, Ribeil JA, Miccio A, Bartolucci P, Leboulch P#, Cavazzana M#. Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial. Nat Med. 2022 Jan;28(1):81-88. (# Corresponding authors)
Magnani A, Semeraro M, Adam F, Booth C, Dupré L, Morris EC, Gabrion A, Roudaut C, Borgel D, Toubert A, Clave E, Abdo C, Gorochov G, Petermann R, Guiot M, Miyara M, Moshous D, Magrin E, Denis A, Suarez F, Lagresle C, Roche AM, Everett J, Trinquand A, Guisset M, Xu Bayford J, Hacein-Bey-Abina S, Kauskot A, Elfeky R, Rivat C, Abbas S, Gaspar HB, Macintyre E, Picard C, Bushman FD, Galy A, Fischer* A, Six E*, Thrasher AJ*#, Cavazzana M*#. Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome. Nat Med. 2022 Jan;28(1):71-80. (*These authors contributed equally; ^#Corresponding authors)
Thompson AA*, Walters MC*, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarré C, Beuzard Y, Chrétien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P*, Cavazzana M*#. Gene therapy for transfusion-dependent β-thalassemia. N Engl J Med. 2018; 378(16):1479-1493. (*These authors contributed equally ^#Corresponding author)
Ribeil JA, Hacein-Bey-Abina S, Payen E, Magnani A, Semeraro M, Magrin E, Caccavelli L, Neven B, Bourget P, El Nemer W, Bartolucci P, Weber L, Puy H, Meritet JF, Grevent D, Beuzard Y, Chrétien S, Lefebvre T, Ross RW, Negre O, Veres G, Sandler L, Soni S, de Montalembert M, Blanche S, Leboulch P#, Cavazzana M#. Gene Therapy in a Patient with Sickle Cell Disease. N Engl J Med. 2017; 376(21):2094 (#Corresponding authors)

PATENTS

2022 BIO21471 Base editing approaches for the treatment of beta-thalassemia- EP22305075

2022 BIO22016 – Methods of inducing cell death of a population of solid tumor cells- EP22305037.8

2022 Methods for assessing the exhaustion of hematopoietic stem cells induced by chronic inflammation – EP22305722.5

2022 Gene Therapy for the treatment of activated PI3kinase delta syndrome type 1 – EP22305686.2

2022 Methods of inducing cell death of a population of solid tumor cells – EP22305037.8

2020 Gene therapy for Mapple Syrup urine disease – PCT/EP2021/054797 – WO/2021/170784

2020 Base editing approaches for the treatment of β-hemoglobinopathies – PCT/EP2021/062633 –

WO/2021/228944

2020 BIO19342 – Base editing approaches for the treatment of β-hemoglobinopathies- EP20305488.7

2020 Bifunctional lentiviral vectors allowing the BS-globin silencing and expression of an antisickling

HBB and uses thereof for gene therapy of sickle cell disease – PCT/EP2021/075887 –

WO/2022/063754

2019 BIO18489 – Optimisation of the scurfy model for in vivo testing of innovative treatments of

autoimmunity- EP19305115.8

2019 Methods for inducing full ablation of hematopoiesis – PCT/EP2020/070244 – WO/2021/009336

2019 Binfunctional vectors allowing BCL11A silecing and expression of an anti-sickling HBB and uses

thereof for gene therapy of β-hemoglobinopathies – PCT/EP2020/057876 – WO/2020/193434

2019 Methods of inducing or restoring immune tolerance – PCT/EP2020/061254 – WO/2020/216807

2019 Recombinant vectors suitable for the treatment of IPEX syndrome (2) – PCT/EP2020/052731 –

WO/2020/161122

2019 Recombinant vectors suitable for the treatment of IPEX syndrome (1) – PCT/EP2019/081820 –

WO/2020/104467

2018 BIO18438 – Methods for inducing full ablation of hematopoiesis (1)- EP18306372.6

2017 APHP-2017-392 – Utilisation de la prostaglandin E2 pour améliorer la survie à long terme des cellules souches hématopoïétiques transduites affectées par leur état inflammatoire- FR1761500

2017 Method for Generating T Cells Progenitors (2) – PCT/EP2018/053406 – WO/2018/146297

2016 A specific trifluoroethyl quinoline analogue for use in the treatment of APDS – PCT/EP2017/061567 – WO/2017/198590

2014 Method for Generating T Cells Progenitors (1) – PCT/EP2015/072889 – WO/2016/055396

CLINICAL TRIALS

Principal Investigator for 7 Phase I/II clinical trials:

EUCT N°2023-507334-24-00: A phase I/II Open Label non-randomized study, monocentric, single arm, evaluating Safety and Efficacy of Gene Therapy of FHL 3 caused by mutations in the human UNC13D gene by transplantation of a single dose of autologous CD34+ cells transduced ex vivo with the UNC13D LV vector expressing the UNC13D cDNA.
EUCT N°: 2024-513652-15-00: A Phase 1/2 Open Label non-randomized Study, multicentric, single arm, evaluating the Safety and Efficacy of Gene Therapy of the severe combined immunodeficiency (SCID) caused by mutations in the human DCLRE1C gene (Artemis) by transplantation of a single dose of autologous CD34+ cells transduced ex vivo with the G2ARTE lentiviral vector expressing the DCLRE1C cDNA
EUCT N°: 2024-516104-42-00: A phase I/II Study evaluating the safety and the efficacy of Human T Lymphoid Progenitor (HTLP) injection to accelerate immune reconstitution after umbilical cord blood (UCB) transplantation in adult patients with hematologic malignancies.
NCT01410019: Gene Therapy for X-linked Severe Combined Immunodeficiency
NCT01347346: Gene Therapy for WAS
NCT02212535: Assessment of Tolerance of Mobilizing Peripheral Hematopoietic Stem Cells by Plerixafor in Sickle Cell Patients
NCT02151526: A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
NCT02633943: Longterm Follow-up of Subjects with Transfusion-Dependent β-Thalassemia Treated With Ex Vivo Gene Therapy
NCT02333760: Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
NCT02813850: Oxygen Therapy and Pregnancy in Sickle Cell Disease

Scientific Director for 4 Phase I/II clinical trials:

START-UP CREATION

SmartImmune : date of creation : July 2017

https://www.smart-immune.com/

Marina Cavazzana is co-founder of SmartImmune and strategic clinical development advisor of the company.

Smart Immune is a clinical-stage biotechnology company developing ProTcell, a thymus-empowered cell therapy platform to fully and rapidly re-arm the immune system, enabling next-generation allogeneic T-cell therapies for all. The company was founded in 2017 to help patients with life-threatening diseases such as high-risk blood cancers and primary immunodeficiencies. Smart Immune’s ProTcell platform, which is already in Phase I/II clinical trials, enables the recovery of a complete immune repertoire in patients fighting cancer and infection. ProTcell introduces potent, allogeneic T-cell progenitors which are then differentiated by the thymus into fully functional T-cells – an ‘off the shelf’ T-cell medicine. Smart Immune’s partners include Memorial Sloan Kettering in New York and Greater Paris University Hospitals (AP-HP).

Smart Immune was co-founded by Marina Cavazzana, Isabelle André and Karine Rossignol at the Imagine Institute in Paris. Based in Paris, in the Paris Biotech Sante Incubator (located within the grounds of Cochin Hospital), Smart Immune has assembled an outstanding group of scientific, technical and clinical experts in lymphoid biology dedicated to changing the future of cellular medicine. Smart Immune has several partnerships with national and international collaborators. We are led by an experienced, multi-disciplinary team with a strong track record of success.

The exclusive ex vivo lymphoid technology of SmartImmune can differentiate hematopoietic stem cells into T cell progenitors in 7 days instead of the usual 15 months needed in human physiology. Once injected into the patient, ProTcell are ableto migrate rapidly to the thymus. The thymus takes just 100 days to ‘educate’ these T cell progenitors, personalizing them to the patient and significantly reducing the time to full immune recovery compared with bone marrow transplantation alone. This is a transformational shift in the benefit/risk ratio of allogeneic T cell medicine. The ProTcell platform has potential for enabling a broad range of future applications including CAR ProTcell and TCR ProTcell for long-lasting, targeted immuno-oncology therapies.

The company received in April 2023 5 miilion investment from Bill and Melinda Gates Foundation. It has also been selected by the European Innovation Council for a funding of €17.5 millions.

RESEARCH GRANTS

2019-2024 Recherche Hospitalo-Universitaire en Santé (RHU-4/IRIS) – Agence Nationale de la Recherche – Title: Gene therapy breakthroughs in inherited immune disorders of the haematopoietic system (Principal Investigator: M. Cavazzana)
2018-2022 H2020-SC1-2017-Two-Stage-RTD (Project #755170 «RECOMB») – Title: Stem-cell based gene therapy for recombination-deficient SCID (Principal Investigator: F. Staal)
2018-2021 E-Rare Net 2018 – Title: Gene edited lymphoid progenitors for adoptive transfer as a treatment of primary immunodeficiency (Principal Investigator: L. Naldini)
2017-2022 Domaine d’Intérêt Majeur (DIM) – Region Ile-de-France – Title: Gene therapy labeled as Area of Major Interest(Principal Investigator: M. Cavazzana)
2016-2022 H2020-ERC-ADG-2015 – ERC-2015 Call for Projects (Project #693762 «GeneForCure») – Title: Expanding and extending gene therapy of monogenic disease of the hematopoietic system (Principal Investigator: M. Cavazzana)
2016-2018 H2020-PHC-2015-two-stage (Project #666908 «SCIDNET») – Title: Developing Genetic Medicines for Severe Combined Immunodeficiency (Principal Investigators: B. Gaspar and F. Staal)
2016–2018 National Agency of AIDS Research (ANRS) – Title: Genotoxicity study of biological characteristics of cord blood derived mononuclear cells in order to evaluate the impact of zidovudine and tenofovir administered to HIV infected pregnant women (Principal Investigator: S. Blanche)
2016–2018 Cancer Research Association (ARC) (Project #PJA20151203306) – Title : Caractérisation de la pathophysiologie du syndrome d’activation PIK3 delta (Principal Investigator : S. Kracker)
2016-2021 Clinical Research Hospital Program (PHRC) (Ministry of Health and Social Affairs) – Title: Acceleration of T cell generation after partially incompatible allogenic HSCT via a brief period of culture on delta-4 chimeric protein (Principal Investigator: M. Cavazzana)
2014–2016 Bluebird Bio – Clinical Research Trial Collaboration – Title: Long-term Follow-up of Subjects treated by ex-vivo gene therapy using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector (Principal Investigator: M. Cavazzana)
2013–2016 National Cancer Institute (INCa) – Title: Study of the action mechanism of oncoprotein TLX: Towards an approach to a differentiating therapy in Acute Lymphoblastic Leukemia ALL-T (Principal Investigator: P. Ferrier)
2013–2016 French Muscular Dystrophy Association (AFM) – Title: Stem cell and T-cell gene therapy using SIN-lentiviral vector in type 3 Familial Hemophagocytic (Principal Investigator: M. Cavazzana)
2013–2015 National League against Cancer (La Ligue contre le Cancer) – Title: Analysis of factors involved in the stability and chromatin remodeling during the process of immunoglobulin isotype switching (Principal Investigator: S. Kracker)
2013-2014 Clinical Research Hospital Program (PHRC) (Ministry of Health and Social Affairs) – Title: The genetic survery of a cohort of patients affected by Common Variable Immunodeficiency (DICV) (Principal Investigators: E. Oksenhendler and S. Kracker)
2013-2018 FP7-HEALTH-2012-INNOVATION-1 Call (Project #305011 «Net4CGD ») – Title: Gene therapy for X-linked chronic granulomatous disease (CGD) (Principal Investigator: A. Galy)
2013-2018 FP7-HEALTH-2012-STREP Call (Project #3054201 «EUROFANCOLEN ») – Title: Phase I/II gene therapy trial of Fanconi anemia patients with a new orphan drug consisting of a lentiviral vector carrying the FANCA gene: a coordinated international action (Principal Investigator: J. Bueren)
2012-2016 National Agency of AIDS Research (ANRS) – Title: Development of a gene therapy protocol for HIV patients affected with hematopoietic malignancies – Acronym “HIV against HIV” (Principal Investigator: M. Cavazzana-Calvo)
2011-2016 FP7-Health – ERC-2010 Call for Projects (Project #269037 « RegenerativeTherapy») – Title: Cell and gene therapy approaches for inherited diseases with unsatisfying or no therapeutic option (Principal Investigator: M. Cavazzana-Calvo)
2011-2015 FP7-HEALTH-2010-single-stage European Call for Projects (Project #261392 «GENEGRAFT ») – Title: Phase I/II ex vivo gene therapy trial for recessive dystrophic epidermolysis bullosa using skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector (Prinicipal Investigator: A. Hovnanian)
2010-2016 FP7-HEALTH-2010-single-stage European Call for Projects (Project #261387 « CELL-PID ») – Title: Advanced cell-based therapies for the treatment of primary immunodeficiency (Principal Investigator: A. Aiuti; Scientific Steering Committee: M. Cavazzana-Calvo, A. Thrasher, G. Wagemaker, C. Baum)
2010-2013 National 2010 Clinical Research Hospital Program (PHRC) (Ministry of Health and Social Affairs) (Project #AOM10013) – Title: Injection of anti-cytomegalovirus and anti-adenovirus CD4+CD8+ T lymphocytes for the treatment of viral infections occurring after allogeneic haematopoietic stem cell transplantation (Principal Investigator: M. Cavazzana-Calvo)
2009-2012 Genethon – Clinical Research Trial Collaboration – Title: Phase I/II clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome (Principal Investigators: A. Fischer, M. Cavazzana-Calvo, Scientific Coordinator: S. Hacein-Bey-Abina)
2009-2011 National 2009 Clinical Research Hospital Program (PHRC) (Ministry of Health and Social Affairs) – Title: Hepatocytes allogeneic transplantation after reversible partial portal embolization to treat congenital defects of the urea cyle (Principal Investigator: I. Dagher, Hôpital Antoine Beclère, Clamart, France)
2009-2011 Medical Research Foundation (FRM) – 2008 Programme « Equipes (Contract n° DEQ20081213950) – Title: Immunotherapy by ex vivo expansion of human T precursor cells following allogenic haematopoietic stem cell transplantation (Principal Investigator: M. Cavazzana-Calvo)
2009-2010 Ministry of Health Hospital Direction (DHOS) / National Institue for Health and Medical Research (INSERM): Call for Projects Funding 2009 – Translational Clinical Research – Title: Set up of a phase I/II clinical trial of ex vivo gene therapy of recessive dystrophic epidermolysis bullosa using grafts of skin equivalents genetically modified with a SIN retroviral vector encoding the COL7A1 cDNA (Principal Investigators: A. Hovnanian, M. Cavazzana-Calvo, S. Hacein-Bey-Abina)
2009-2010 National Agency of AIDS Research (ANRS) – Title: Persistant alteration of haematopoiesis in children after in utero AZT-3TC exposure (Principal Investigator: S. Blanche)
2008-2011 National 2008 Clinical Research Hospital Program (PHRC) (Ministry of Health and Social Affairs) – Title: Protocol n.2 of gene therapy for X-linked severe combined immunodeficiency (SCID-X1) using a secured retroviral vector (project AOM 08 064) (Principal Investigators: M. Cavazzana-Calvo, A. Fischer)
2008-2010 Ministry of Health Hospital Direction (DHOS) / National Institute for Health and Medical Research (INSERM) / National Institute for Cancer (INCa): Call for Projects Funding 2008 – Translational Clinical Research – Title: Development of an ex vivo expansion method for T lymphoid progenitors by short exposition to the Noth ligand to try to accelerate the immunological reconstitution after allogenic hematopoietic stem cell transplantation using a partially HLA compatible donor (Principal Investigators : M. Cavazzana-Calvo, I. André-Schmutz)
2008-2010 National Agency for Research (ANR) – Biological Innovation Network – Title: Development of tetramers/CMH and of methods of selection compatible with their clinical use in adoptive immunotherapy against CMV (Convention n. ANR-07-RIB-018-02) (Principal Investigator: V. Monchois, ProteinXpert)
2006–2010 National Institute for Health and Medical Research (INSERM) (Promotor) Biomedical Research Project – Title : Traitement des formes cérébrales d’adrénoleucodystrophies liées à l’X de l’enfant par transfert ex vivo du gène dans les cellules CD34+ autologues (protocole RBM 03 38 – convention 06-21-003). (Principal Investigator: Pr. Aubourg, St Vincent de Paul)
2006–2009 Specific Targeted Research Project – European research contract INTEGRA – Title: New tools for genetic engineering using targeted integration vectors: application to agronomy, food safety and gene therapy (Principal Investigator: J Mallet)
2006–2008 National Agency for Research (ANR) – 2005 Program on Rare Diseases: SCID Gene Therapy – Title: Gene therapy of severe combined immunodeficiency caused by mutations of the γc, Artemis and RAG1 genes (Principal Investigator: M. Cavazzana-Calvo)
2005-2011 Genetix Pharmaceuticals – Clinical Research Trial Collaboration – Title: A phase I-II open label study with anticipated clinical benefit evaluating genetic therapy of the B-hemoglobinopathies by transplantation of autologous CD34+ stem cells modified ex-vivo with a lentiglobin vector (contract LG001 – last amendment 21/08/08) (Principal Investigator: M. Cavazzana-Calvo)
2005–2007 National Agency for Research (ANR) – Biological Innovation Network – Title: Therapy of severe combined immunodeficiencies (SCID) by DNA surgery (Principal Investigator: D. Sourdive, CELLECTIS S.A.)
2004–2007 European Consert Contract “Concerted Safety & Efficiency” – Title: Evaluation of Retroviral Transgenesis in Gene Therapy of Inherited Diseases (Contract No. LSHB-CT-2004-005242) (Principal Investigator: G. Wagemaker)
2004–2005 National Clinical Research Hospital Program (PHRC) (Ministry of Health and Social Affairs) on Gene Therapy – Title: Treatment of X-linked severe combined immunodeficiency (γc deficiency) by ex-vivo transfer of gene γc in bone marrow cells CD34+ autologues (Principal Investigator: M. Cavazzana-Calvo)
2003–2006 Biotherapy Concerted Action – “Stem Cells” invitation to tender – Title: Common Lymphoid Precursors (Principal Investigator: S. Ezine)
2003–2006 French Muscular Dystrophy Association (AFM) – Title: Clinical and preclinical investigations of severe combined immunodeficiencies (SCID) caused by recombined V(D)J defects (Principal Investigator: A. Fischer)
2002–2004 National Clinical Research Hospital Program (PHRC) (Ministry of Health and Social Affairs) Alloreactive Depletion (Ministry of Health and Social Affairs) – Title: Grafting of purified hematopoietic stem cells in CD34+ cells to children with non-genetically identical histocompatibility: prevention of graft-versus-host reaction and acceleration of immune reconstitution by transfusion of T lymphocytes from donors with no alloreactive response against the recipient. (Principal Investigator: M. Cavazzana-Calvo)
2002–2004 European Consortium of Researchers (INHERINET contract No. QLK3-CT2001-00427) – Title: Gene therapy of hematopoietic stem cells for inherited diseases (European Coordinator: G. Wagemaker)
2002 French Muscular Dystrophy Association (AFM) – Title: Alloreactive Depletion (Principal Investigator: M. Cavazzana-Calvo)
2002 French Muscular Dystrophy Association (AFM) – Title: Program study of chromosome insertion sites of retrovirus and their frequences (Principal Investigator: A. Fischer)
2000–2002 European Consortium of Researchers (Contract No. QLK3-1999-00859) – Title: Lentiviral vectors for gene therapy of the hematopoietic system: development from bench to bedside (Principal Investigator: L. Naldini)
2000 National Clinical Research Hospital Program (PHRC) (Ministry of Health and Social Affairs) (Project code: AOM00093) – Title: Injection of anti-EBV CTL for the prevention of EBV-induced lymphoproliferative syndromes after partially compatible bone marrow grafts (Principal Investigator: M. Cavazzana-Calvo)
2000 Ministry of Research – Title: Usage of genoplasty in the treatment of immune deficiencies diseases (Principal Investigator: M. Cavazzana-Calvo)
2000 Post-Genome-Genomic Sequencing Action II with the Ministry of Research – Title: Preclinical evaluation of Il-7 in a model of immune reconstitution. Study performed prior to clinical development of an orphan indication (Principal Investigator: M. Cavazzana-Calvo)
2000 French Muscular Dystrophy Association (AFM) – Title: Development of the therapeutic potential of stem cells for cell therapy in the treatment of hereditary diseases (Principal Investigator: M. Cavazzana-Calvo)
2000 National League against Cancer (La Ligue contre le Cancer) – Title: Generation of specific cytotoxic T lymphocytes with a view to treatment of the Epstein-Barr virus-induced lymphoproliferative B syndrome after bone marrow and organ transplant (Principal Investigator: A. Fischer)
2000 National League against Cancer (La Ligue contre le Cancer) – Title: Complement used to adapt the automatic reading system on 96-well microtest plates using the ELISPOT technique (Principal Investigator: M. Cavazzana-Calvo)
2000 National Clinical Research Hospital Program (PHRC) (Ministry of Health and Social Affairs) – Title: Gene therapy of severe combined immune deficiencies (Principal Investigator: A. Fischer)
2000 French Muscular Dystrophy Association (AFM) – Title: Gene therapy of severe combined immune deficiencies (Principal Investigator: A. Fischer)
2000 National Agency of AIDS Research (ANRS) – Title: Assessment of the role of interleukin-7 in the acceleration of immune reconstitution (Principal Investigator: N. Israel)
1999 Ministry of Health Hospital Direction (DHOS) / National Institute for Health and Medical Research (INSERM) / National Institute for Cancer (INCa) – Title: Determination of the optimal dose of G-CSF after chemotherapy with a view to collecting blood stem cells for autografts (Principal Investigator: F. Lefrère)
1999 French Blood Agency (EFS) – Title: Gene therapy of severe combined immune deficiencies and optimization of gene transfer into hematopoietic cells (Principal Investigator: M. Cavazzana-Calvo)
1998 National Institute for Health and Medical Research (INSERM) PROGRES project – Title: Anti-infectious, cytotoxic lymphocytes (Principal Investigator: H. Vié)
1997 Fondation de France – Title: Anti-EBV cytotoxic lymphocytes (Principal Investigator: M. Cavazzana-Calvo)
1997 Clinical Research Management (DRC-AP-HP): cell therapy call for tender – Title: Antiviral Immune cell therapy (Principal Investigator: M. Cavazzana- Calvo)
1996 National Clinical Research Hospital Program (PHRC) (Ministry of Health and Social Affairs) – Title: Gene therapy of severe combined immune deficiencies (Principal Investigator: A. Fischer)
1995 National Clinical Research Hospital Program (PHRC) (Ministry of Health and Social Affairs) – Title: Alloreactive T depeletion to prevent graft rejection and graft versus host reaction after genetically identical non-HLA bone marrow allogenic grafting (Principal Investigator: M. Cavazzana- Calvo)
1994 French Blood Agency (EFS) – Title: Gene therapy of X-linked Severe Combined Immune Deficiency and adenosine deaminase deficiency (Principal Investigator: M. Cavazzana- Calvo)

SCIENTIFIC AWARDS

2022 European Hematology Association (EHA)-José Carreras Award
2021 Cell Therapy Transplant Canada (CTTC)-2021 Fred Sanders Lectureship Award
2019 University of Zurich Dies Academicus Prize – Honorary Doctorates
2017 Ernest Beutler Lecture and Prize for Clinical Science (American Society of Hematology)
2017 Fondation Guillaume-Piel Prize from the Medical Research Foundation
2017 Innovation Prize 2017 – Bioentrepreneurs T-rex Bio Program
2016 Prize of the National Academy of Medicine
2013 Woman of Excellence Award – Padua, Italy
2012 Irène Joliot Curie Prize of the “Femme Scientifique de l’Année” awarded by the Academy of Sciences and the French Ministry of Higher Education and Research
2011 Title of Officer of the National Order of the Legion of Honor
2009 Prize for Exceptional Achievements of the European Society of Gene and Cell Therapy
2008 Matmut Prize for Innovation (3rd Foundation of the Future Trophy)
2007 INSERM Prize 2006 for Clinical and Therapeutic Research
2004 Jean-Pierre Lecocq Prize in Gene Therapy – French Academy of Sciences
2001 Title of Knight of the National Order of the Legion of Honor
2000 Medical Prize from the Toulouse Academy of Sciences
1999 Prize of the American Society of Hematology for clinical research in Gene Therapy
1999 Philip Morris Scientific Prize on Gene Therapy
1997 Prize from the Medical Research Foundation (Clinical Research)

NATIONAL APPOINTMENTS AND COLLECTIVE RESPONSIBILITIES

Elected member – National Academy of Medicine – November 2023
4th National Rare Diseases Plan (PNMR) – Lead of subgroup « thérapies innovantes » (Oct 2023)
Consultancy for the revision of the Biotherapic Law n°2011-814 study with a view to a revision by the Parliament (March, 5^th 2018)
Coordinator of the DIM Gene Therapy (2017-2022)
Member of the Scientific Committee of the “Association de Patients Déficits Immunitaires Primitifs” IRIS (2014-Present)
Member of the Administration Council of l’Envol, “The campus of La Banque Postale” (2013-Present)
Vice-president of the Observatoire International de la Drépanocytose (2012-Present)
Member of the Clinical Research Commission, University Paris Descartes-Sorbonne Paris Cité (2010-2012)
Member of EUROCORD Association (2010-Present)
Member of the Academic judging committee to the nomination of the Biotherapy Chair, University Paris Descartes (2010)
Vice-President of the Scientific Board of the Foundation for Medical Research (FRM) (2010)
Member of the Scientific Advisory Board of Genethon (2008-2012)
Chairwoman of the Scientific Committeefor Strategies and Evaluation of Clinical Research Protocols(INSERM) (2006 – 2011)
Member of the French Muscular Dystrophy Association (AFM) Commission for clinical trials & of AFM’s Scientific Committee (2002-2008)
Biotherapy Consortium of Researchers Member, INSERM. Responsible for the Stem Cell Sub-committee (2001)
Member of the National French Cancer Research Association Commission No.1 (2000-2005)
Vice chairwoman of INSERM intercommission No.5 (2000-2002)
President of the Therapeutic Innovation Research Commission of Hospices Civiles de Lyon Clinical Research Direction (2000-2002)
Member of the consortium of researchers group No. 21 (“Constituted lymphocyte hemostasis”) for National Agency of AIDS Research (ANRS) (1998-2000)
Expert with the French Agency for the Safety of Health Products (1997-2010)
Member of the Scientific Board of the French Society of Bone Marrow Graft (1996-2000)
Member of the scientific committee “French Society of Gene and Cell Therapy” (1996-2010)

INTERNATIONAL APPOINTMENT AND COLLECTIVE RESPONSABILITIES

Co-chair ASH-SITC Scientific Workshop – 2025 ASH Annual Meeting
Chair – EBMT 2025 – Session title: ATMPs
Chair of Scientic SJD Evaluators – IRSJD EVALUATION -November 27th 2024
Chair of the ASH subcommittee’s initiative on Gene Therapy for Classical Hematologic Diseases (april 2024-present)
Chair of ASH’s SCD Research Priorities Working Group as a Working Group Leader (feb. 2024-present)
Member of Editorial Board of International Journals
Member of the National Academy of Medecine (2019)
Member of the International Advisory Board of Lancet Hematology (2016-Present)
Specialty Chief Editor of Innovative Therapies of Frontiers in Medicine (2016-Present)
Member to the Editorial Board of the journal Regenerative Engineering and Translational Medicine (2015-Present)
Member of the Editorial Board of the jounal Cell and Gene Therapy Insights (2015-Present)
Member of the Committee of Encyclopedia of Immunology 3^rd Edition (2014)
Member of the Editorial Board of the jounal Molecular and Cellular Pediatrics (2013-Present)
Member of the Editorial Board of Stem Cells Translational Medicine (2011-Present)
Blood Editorial Board Invited Member (2008-2012)

Membership in the Scientific Societies and Scientific Responsabilities

Member of the Henry Kunkel Society (HKS) – 19 janvier 2024-present

Keystone Symposim

Scientific Organizer of the “Emerging Cellular Therapies: Cancer and Beyond” Congress, joint with Engineering the Genome (Canada, 2020)

American Society of Hematology (ASH)

American Society of Hematology (ASH) (2016)

Member of the Scientific Committe of the American Society of Hematology (ASH) at the 56^th Annual Meeting (2014-2015)
American Society of Hematology (1996-Present)

American Society for Gene and Cell Therapy (ASCGT)

Member of the American Society of Gene & Cell Therapy (ASGCT) Advisory Council (2015-Present)
Member of the Board of Directors of the American Society for Gene and Cell Therapy (ASGCT) (2011-2014)
American Society of Gene Therapy (1996-Present)

European Research Council (ERC)

Lead reviewer for European Research Council Consolidator Grants Evaluation (ERC-2015-CoG) Panel LS7: 101 (2015)
Member of the Consolidator Grant Panel LS7C “Diagnostic Tools, Therapies and Public Health” for the European Research Council (ERC) (2013)

Other Scientific responsabilities

Member of the Novel Approaches to Hematological Diseases (NAHD) scientific advisory committee (2018)
Member of UNAIDS Scientific Expert Panel (2013-2015)
Member of the Scientific Committee of the Cansearch Foundation (2012-2021)
Invited Member of the Andalusian Initiative for Advanced Therapies (AIATA) Education Advisory Board (2011)
Member of the Scientific Board of the European CONSERT Advanced Course Symposium and Practical Training on “Lentiviral vectors: concepts, practice, hope and reality”; Chairwoman of the 2008 Course (2008)
Member of the taskforce for the writing of ethical guidelines about the clinical use of embroyonary stem cells (2008-2010)
Member of the American Society of Gene Therapy Hematopoietic Committee & of the Ethics Committee (2006-2008)
Inborn Errors Working Party Chairwoman & European Society for Blood and Marrow Transplantation (EBMT) Group Scientific Council Member (2001-2008)
Member of the Transatlantic Gene Therapy Consortium (2000-2005)
Member of the European Society for Gene & Cell Therapy (1999-Present)
Member of the International Society for Hemotherapy and Graft Engineering (1995-2000)
Member of the Pediatric Hematology and Immunology Society (1993-Present)

ACADEMIC TEACHING

Sorbonne university – Paris Saclay University

IUD Ethics in Procreation and Perinatality: Postnatal screening in the general population. Monday, June 3, 2024
Stem Cell Biology, Master’s Program, 2^nd year: Gene therapy of genetic disease of the hematopoietic system, September 27, 2023

Paris Descartes University, Faculty of Medecine

Cell Therapies, Master in Cell Biology, Development and Reproduction, 2013-2016
Research in Gene Therapy, Master in Ethics Research, 2013-2022
Genetic, Cellular and Clinical Bases of immunodeficiencies and of inherited hyper-immune syndromes, CG2ID Doctoral School, 2011-2017
Medical Genetics, Master in Health, 2005
Biology of Stem Cells, Master in Molecular & Cellular Biology, 2002-2015
Medical Genetics, Master in Molecular Genetics of Disease Development and Oncogenesis (Dir: Professor P. Aubourg), 2002-2008
Human Genetics, Master of Medical and Biological Science, 2002-2004

Other training activities

Using gene therapy for the hereditary diseases of the hematopoietic system, 2^ndEuropean MD-PhD Conference, Association Medecine Pharmacie Sciences, Paris, 2013
Immune Regeneration, Gene therapy for primary immunodeficiencies. Hanover Biomedical Research School Master Class, Hanover Medical School, Germany, 2012
Molecular & Cell Biotherapies Master in Biomedical Engineering, Paris Tech and Paris-Descartes University (Dir: Pr. S. Hacein-Bey-Abina), 2011-Present
Master in Advanced Immunology, Pasteur Institute, Paris V, VI, VII University, 2011-Present
Courses at the School of INSERM, 2010-Present
Master in Therapeutics & Biotechnology, Paris VII University (Dir: T. Saison-Behmoaras), 2007-2010
Master in Experimental and Applied Pharmacology, Paris VII University (Dir: Pr. Calvo – Pr. Jacqz), 2005-2008
Master in Fundamental Virology, Pasteur Institute, 2002-Present

Before 2005

Continuous Education of the Teaching Academy of Versailles, Biochemistry & Biology Department (Chairman: Pr. Sabine Orsoni)
The Hesperis Course, European Society for Organ Transplantation Education Program (Chairman: P. Burra, C. Legendre)
University Diploma in Immunology and Blood Transfusion, Pierre & Marie Curie Paris V University (Dir: Pr. Rouger)
University Diploma in Cell Therapy, Paris VII University (Dir: Pr. Chomienne)
University Diploma in Onco-Hematology (Dir: Pr. Bierling)
University Diploma in Transfusion Medicine, Broussais Hospital – Hôtel Dieu, Paris (Dir: Pr. Baron, Pr. Rouger)
University Diploma in Immunological Treatments, Paris VI University (Dir: Pr. W.H. Fridman)

SUPERVISION

Postdoctoral fellows’ supervision

Tayebeh Shabahang-Soheili (Iranian) MUNC 13-4 gene therapy project 2011-2017
Laura Simons (German) Generation of T-cell precursors from adult HSC 2014-2015
Study of human thymic niche 2010-2015

Farid Ouchani Characterization of moesin deficiency 2011-2013
Julia Hauer (German) Role of RAG1 in the development of B-ALL 2006-2009
Emmanuelle Six Postnatal human lymphopoiesis 2005-2008
Recruited by INSERM as researcher in 2009
Fatine Benjelloun Artemis gene therapy project 2004-2007
Chantal Lagresle RAG1 gene therapy project 2002-2007
Recruited as Research Engineer by INSERM in 2009
Isabelle André-Schmutz Cell therapy of immune deficiency 2000-2002
Recruited by INSERM as researcher in 2002
Ability to Lead a Research obtained in 2010
Recruited by INSERM as research director in 2012, co-head of the lab since 2014

Thesis supervision
Jean-Sebastien Diana First live track detection of HIV DNA in humanized model by single-cell imaging 2019-2022

Giulia Hardouin Développement d’une nouvelle approche de thérapie génique pour la drépanocytose par édition de base 2018-2022

Steicy Sobrino Clonal mapping and gene correction of hematopoietic stem cells 2018-2022
Marianne Delville Gene therapy for IPEX syndrome 2016-2019
Leslie Weber ne therapy of red blood cell diseases 2015-2018
Kuiying Ma (Chinese) Role of Notch pathway in the generation of T-cell progenitors 2014-2018
Hicham Lamrini (Morrocan) Identification and description of new molecular causes of Ig CSR-D and hypogammaglobulinemia 2014-2018
Lucie Heurtier Congenital hyperactivation of the P13K pathway: a new cause of immunodeficiency 2014-2017
Andrea Schiavo (Italian) Study of lymphoid progenitors circulating after mobilization with plerixafor 2010-2011
Co-Tutorship Agreement between Padua (Italy) and Paris-Descartes Universities
Christian Reimann (German) In vitro generation of potent T-Lymphoid progenitors in a feeder-cell-free DL-4 sytem 2009-2012
Andrea Ditadi (Italian) Cell therapy approach for haematopoietic diseases using fetal cells issued from amniotic fluid 2004-2008
Co-Tutorship Agreement between Padua (Italy) and Paris-Descartes Universities
Yamina Hamel Anti-infectious, adoptive immunotherapy 1999-2002
Frank Yates Gene therapy of RAG2 deficiency 1999-2003
Salima Hacein-Bey Gene therapy in X-SCID 1995-1998
Recruited as Hospital Practitioner by Necker-Enfants Malades Hospital in 1999
Recruited as Hospital Practitioner and University Lecturer by Paris-Descartes University in 2008

Master student supervision

Lounes Djerroudi 2013-2014
Elodie Elkaïm 2013-2014
Hicham Lamrini 2013-2014
Lucie Heurtier 2013-2014
Alexandre Vivanti 2012-2013
Sarah Lechat 2011-2012
Kevin Appourchaux 2011-2012
Eva-Maria Weick (German) 2008-2009
Allen Liu (American) 2003-2004
Danielle Bensoussan 1995-1996
Juliette Lemerle 1993-1994
Catherine Boccaccio 1992-1993
Elie Haddad 1991-1992
Jean-Louis Stephan 1990-1991
Dominique Valteau-Couanet 1988-1989

Other supervision activities

Julie Rivière, Engineer Diploma, National Conservatory of Arts and Crafts (CNAM, Paris) (2006-2014)
Christophe Hue, Engineer diploma, Ecole Pratique des Hautes Etudes (EPHE, Paris) (2001-2003)
Eric Yvon, Engineer diploma, Ecole Pratique des Hautes Etudes (EPHE, Paris) (1996-1998)

Member of the defense jury

Megane Brusson-HDR – Medecine-From red blood cell exploration to therapeutic innovations for beta-hemoglobinopathies (June 19, 24)
Robert Janie – Doctor of Philosophy in Molecular and Cell Biology- ED394 Ecole doctorale Physiologie, Physiopathologie, Thérapeutique Laboratory Immunology-Immunopathology-Immunotherapy (i3) UMRS959 (Sorbonne Université) – Interleukin-2-self-sufficient regulatory T cells for the treatment of autoimmune and inflammatory diseases (28/06/24)
Cristina Claver– Doctor of Pharmacy – What market access strategies should be adopted for innovative therapy drugs? Comparative analysis of European and American markets: limitations, challenges, and trends

Activities reporter

Gina Jimenez, Doctoral Thesis in Sciences, Lyon 1 University, “Gene Therapy in Primary Ciliary Dyskinesia” (2013)
Lien De Somer, Doctoral Thesis in Sciences, Catholic University of Leuven, Belgium, “Novel approaches to balance graft-versus-leukemia and graft-versus-host immune effects of allogeneic hematopoietic stem cell grafts: studies in mice” (2010)
Lidia Sinka, Doctoral Thesis in Blood Cell Biology, Paris-Diderot VII University, “Angiotensin converting enzyme in the blood system ontogeny of the human embryo” (2008)
Ibrahim Dagher, Ability to Lead a Research, Paris-Sud XI University, “Hepatocyte transplantation in murine and primate models: study of factors improving engraftment” (2008)
Vanderson Rocha, Ability to Lead a Research, Paris- Diderot VII University, “Ombilical Cord, peripheral blood and bone marrow hematopoietic stem cells graft: study of risks factors related to patients’ diseases, donors and immunogenetics” (2007)
Danièle Bensoussan-Lejzerowicz, Bioengineering Doctoral Thesis, Henri Poincaré Nancy I University, “Congenital defects of cell immunity and cell therapy: usefullness of cell therapy to prevent complications after hematopoietic stem cell allografts” (2007)
Oumeya Adjali, Doctoral Thesis, Montpellier II University, “Modulation de la différenciation des cellules T par thérapies cellulaire et génique ciblant le thymus : module du déficit immunitaire lié à l’absence de ZAP-70” (2006)
Sébastien Boni, Microbiology Doctoral Thesis, Paris VI University, “Observation de la modulation de l’activité traductionnelle de l’IRES du virus de l’hépatite C par certains facteurs viraux, et mise au point d’un système d’étude cellulaire de son activité via un vecteur lentiviral” (2006)
Rima Haddad, Doctoral Thesis, Paris VII University “Biology of Blood Cells” (2005)
Dominique Mahieu-Caputo, Doctoral Thesis, Paris V University, “Fetal hepactocytes, potential vectors for cell therapy in hepatic diseases” (2004)
Delphine Sauce, Doctoral Thesis, Faculty of Medecine and Pharmacy, Besançon, Franche Comté University, “Impact of the gene transfer process on the phenotype and functional properties of T lymphocytes genetically modified in bone marrow graft” (2003)

PUBLICATION LIST

Original Publications in Scientific Journals

Original Articles in Scientific international Journals

Sobrino S, Joseph L, Magrin E, Chalumeau A, Hebert N, Corsia A, Denis A, Roudaut C, Aussel C, Leblanc O, Brusson M, Felix T, Diana JS, Petrichenko A, El Etri J, Godard A, Tibi E, Manceau S, Treluyer JM, Mavilio F, Bushman FD, Marcais A, Castelle M, Neven B, Hermine O, Renolleau S, Magnani A, Asnafi V, El Nemer W, Bartolucci P, Six E, Semeraro M, Miccio A, Cavazzana M. Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease. Nat Commun. 2025 Apr 1;16(1):3137. doi: 10.1038/s41467-025-58321-4. PMID: 40169559; PMCID: PMC11961595.
Corsia A, Joseph L, Beeker N, Manceau S, Driessen M, Meunier B, Cavazzana M, Collier M, Treluyer JM. Maternal and perinatal outcomes of sickle cell disease in pregnancy: A nationwide study in France. Br J Haematol. 2025 Feb 21. doi: 10.1111/bjh.20009. Epub ahead of print. PMID: 39984807.
Garrigue A, Kermasson L, Susini S, Fert I, Mahony CB, Sadek H, Luce S, Chouteau M, Cavazzana M, Six E, Le Bousse-Kerdilès MC, Anginot A, Souraud JB, Cormier-Daire V, Willems M, Sirvent A, Russello J, Callebaut I, André I, Bertrand JY, Lagresle-Peyrou C, Revy P. Human Oncostatin M deficiency underlies an inherited severe bone marrow failure syndrome. J Clin Invest. 2025 Jan 23:e180981. doi: 10.1172/JCI180981. Epub ahead of print. PMID: 39847438.
Río P, Zubicaray J, Navarro S, Gálvez E, Sánchez-Domínguez R, Nicoletti E, Sebastián E, Rothe M, Pujol R, Bogliolo M, John-Neek P, Bastone AL, Schambach A, Wang W, Schmidt M, Larcher L, Segovia JC, Yáñez RM, Alberquilla O, Díez B, Fernández-García M, García-García L, Ramírez M, Galy A, Lefrere F, Cavazzana M, Leblanc T, García de Andoin N, López-Almaraz R, Catalá A, Barquinero J, Rodríguez-Perales S, Rao G, Surrallés J, Soulier J, Díaz-de-Heredia C, Schwartz JD, Sevilla J, Bueren JA; FANCOLEN-1 gene therapy investigators. Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials. Lancet. 2025 Dec 21;404(10471):2584-2592. doi: 10.1016/S0140-6736(24)01880-4. Epub 2024 Dec 3. PMID: 39642902.
Doye A, Chaintreuil P, Lagresle-Peyrou C, Batistic L, Marion V, Munro P, Loubatier C, Chirara R, Sorel N, Bessot B, Bronnec P, Contenti J, Courjon J, Giordanengo V, Jacquel A, Barbry P, Couralet M, Aladjidi N, Fischer A, Cavazzana M, Mallebranche C, Visvikis O, Kracker S, Moshous D, Verhoeyen E, Boyer L. RAC2 gain-of-function variants causing inborn error of immunity drive NLRP3 inflammasome activation. J Exp Med. 2024 Oct 7;221(10):e20231562. doi: 10.1084/jem.20231562. Epub 2024 Aug 30. PMID: 39212656; PMCID: PMC11363864.
Sorel N, Díaz-Pascual F, Bessot B, Sadek H, Mollet C, Chouteau M, Zahn M, Gil-Farina I, Tajer P, van Eggermond M, Berghuis D, Lankester AC, André I, Gabriel R, Cavazzana M, Pike-Overzet K, Staal FJT, Lagresle-Peyrou C. Restoration of T and B Cell Differentiation after RAG1 Gene Transfer in Human RAG1 Defective Hematopoietic Stem Cells. Biomedicines. 2024 Jul 5;12(7):1495. doi: 10.3390/biomedicines12071495. PMID: 39062069; PMCID: PMC11275127.
Moiani A, Letort G, Lizot S, Chalumeau A, Foray C, Felix T, Le Clerre D, Temburni-Blake S, Hong P, Leduc S, Pinard N, Marechal A, Seclen E, Boyne A, Mayer L, Hong R, Pulicani S, Galetto R, Gouble A, Cavazzana M, Juillerat A, Miccio A, Duclert A, Duchateau P, Valton J. Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells. Nat Commun. 2024 Jun 11;15(1):4965. doi: 10.1038/s41467-024-49353-3. PMID: 38862518; PMCID: PMC11166989.
Below are another 260 publications.

…………………………………….

Didactic Publications

Original Reviews and Editorials in Scientific International Journals

Cavazzana M#, Corsia A, Brusson M, Miccio A, Semeraro M. Treating Sickle Cell Disease: Gene Therapy Approaches. Annu Rev Pharmacol Toxicol. 2024 Sep 11. doi: 10.1146/annurev-pharmtox-022124-022000. Epub ahead of print. PMID: 39259977. (#corresponding author)
Cavazzana M#, Calvo C. A new step toward non-genotoxic conditioning prior to hematopoietic stem cell transplantation. Mol Ther. 2024 May 21: S1525-0016(24)00327-7. doi: 10.1016/j.ymthe.2024.05.025. Epub ahead of print. PMID : 38776908. .( #corresponding author)
Locatelli F, Cavazzana M, Frangoul H, Fuente J, Algeri M, Meisel R. Autologous gene therapy for hemoglobinopathies: From bench to patient’s bedside. Mol Ther. 2024 May 1;32(5):1202-1218. doi: 10.1016/j.ymthe.2024.03.005. Epub 2024 Mar 7. PMID: 38454604; PMCID: PMC11081872.
Cavazza M#, Miccio A. The difficult translational pathway from animal models to patients. Cell Stem Cell. 2024 Apr 4 ;31(4) :435-436. Doi : 10.1016/j.stem.2024.03.010. PMID : 38579680. .( #corresponding author)
Hardouin G, Magrin E, Corsia A, Cavazzana M, Miccio A, Semeraro M. Sickle Cell Disease: From Genetics to Curative Approaches. Annu Rev Genomics Hum Genet. 2023 Aug 25;24:255-275. doi: 10.1146/annurev-genom-120122-081037. PMID: 37624668.
Abou-El-Enein M, Angelis A, Appelbaum FR, Andrews NC, Bates SE, Bierman AS, Brenner MK, Cavazzana M, Caligiuri MA, Clevers H, Cooke E, Daley GQ, Dzau VJ, Ellis LM, Fineberg HV, Goldstein LSB, Gottschalk S, Hamburg MA, Ingber DE, Kohn DB, Krainer AR, Maus MV, Marks P, Mummery CL, Pettigrew RI, Rutter JL, Teichmann SA, Terzic A, Urnov FD, Williams DA, Wolchok JD, Lawler M, Turtle CJ, Bauer G, Ioannidis JPA. Evidence generation and reproducibility in cell and gene therapy research: A call to action. Mol Ther Methods Clin Dev. 2021 Jul 21;22:11-14.
Moreno-Corona N, Chentout L, Poggi L, Thouenon R, Masson C, Parisot M, Mouel LL, Picard C, André I, Cavazzana M, Perrin L, Durandy A, Azarnoush S, Kracker S. Two Monogenetic Disorders, Activated PI3-Kinase-δ Syndrome 2 and Smith-Magenis Syndrome, in One Patient: Case Report and a Literature Review of Neurodevelopmental Impact in Primary Immunodeficiencies Associated With Disturbed PI3K Signaling. Front Pediatr. 2021 Jun 24; 9:688022.
André I, Simons L, Ma K, Moirangthem RD, Diana JS, Magrin E, Couzin C, Magnani A, Cavazzana M. Ex vivo generated human T-lymphoid progenitors as a tool to accelerate immune reconstitution after partially HLA compatible hematopoietic stem cell transplantation or after gene therapy. Bone Marrow Transplant. 2019 Aug;54(Suppl 2):749-755.
Cavazzana M, Bushman FD, Miccio A, André-Schmutz I, Six E. Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges. Nat Rev Drug Discov. 2019 Jun;18(6):447-462.
Simons L, Cavazzana M, André I. Concise Review: Boosting T-Cell Reconstitution Following Allogeneic Transplantation-Current Concepts and Future Perspectives. Stem Cells Transl Med. 2019 Jul;8(7):650-657.
Cavazzana M. Four decades of progress. Blood. 2022 Aug ; 140 (7): 665–666.
Cavazzana M#, Thrasher A. Gene therapy for Whiskott-Aldrich syndrome: The latest news. Clin Transl Med. 2022 Apr;12(4):e815. .( #corresponding author)
Cavazzana M#, Mavilio F. Gene Therapy for Hemoglobinopathies. Hum Gene Ther. 2018 Oct;29(10):1106-1113.( #corresponding author)
Missontsa MM, Bernaudin F, Fortin A, Dhédin N, Pondarré C, Yakouben K, Neven B, Castelle M, Cavazzana M, Lezeau H, Peycelon M, Paye-Jaouen A, Sroussi J, Diesch-Furlanetto T, Barraud-Lange V, Sarnacki S, Fahd M, Marchand I, Delcour C, Vexiau D, Arlet JB, Kamdem A, Arnaud C, Dalle JH, Poirot C. Ovarian tissue cryopreservation for fertility preservation before hematopoietic stem cell transplantation in patients with sickle cell disease: safety, ovarian function follow-up, and results of ovarian tissue transplantation. J Assist Reprod Genet. 2024 Feb 15. doi: 10.1007/s10815-024-03054-4. Epub ahead of print. PMID: 38358434.
Staal FJT, Aiuti A, Cavazzana M. Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives. Front Pediatr. 2019 Oct 31;7:443.
Below are another 77 publications ……………………………..

Original Reviews in Scientific National Journals

Cavazzana M. Gene therapy: many more questions than answers. Médecine Sciences. 2017 May;33(5):461-462. doi: 10.1051/medsci/20173305001. Epub 2017 Jun 14.
Ribeil JA, Blanche S, Cavazzana M. Gene therapy for sickle cell disease. Médecine Sciences. 2017 May;33(5):463-465. doi: 10.1051/medsci/20173305002. Epub 2017 Jun 14
Galy A, Corre G, Cavazzana M, Hacein-Bey-Abina S. Efficacité et sécurité du traitement par thérapie génique des patients atteints du syndrome de Wiskott-Aldrich. Médecine Sciences. 2015 Dec;31(12):1066-1069.
Cavazzana-Calvo M. La thérapie génique : où en est-on ? Rev Prat. 2013 ; 63:457-61.
Cartier N, Hacein-Bey-Abina S, Von Kalle C, Bougnères P, Fischer A, Cavazzana-Calvo M, Aubourg P. Gene therapy of X-linked adrenoleukodystrophy using hematopoietic stem cells and a lentiviral vectorBull Acad Nalt Med. 2010 ; 194:255-268.
Cavazzana-Calvo M, Hacein-Bey-Abina S, Fischer A. Ten years of gene therapy: thoughts and perspectives. Médecine Sciences. 2010; 2:115-118.
Cavazzana-Calvo M,. 20 ans de thérapie génique. Biofutur. 2009 ; 300 : 47-8.
Mahlaoui N,Cavazzana-Calvo M. Guérir un déficit immunitaire congénital, Rev Prat. 2007 ; 57 : 1699-1700.
Cavazzana-Calvo M, Dal-Cortivo L, André-Schmutz I, Hacein-Bey Abina S, FischerA. Cell therapy for inherited diseases of the hematopoietic system. C R Biol. 2007; 330: 538-42.
Cavazzana-Calvo M, Six E, Andre-Schmutz I, Coulombel L. Human hematopoiesis: from CD34 cells to T lymphocytes. Médecine Sciences. 2007; 23:151-160.
Six E, Andre Schmutz I, Cavazzana-Calvo M. Notch ligands Delta and lymphoid development niches.Médecine Sciences. 2007; 23:21-4.
Fischer A, Hacein-Bey-Abina S, Lagresle C, Garrigue A., Cavazzana-Calvo M. Thérapie génique des deficits immunitaires sévères : prevue de principe d’efficacité et problèmes soulevés. Bull Acad Natl Méd. 2005; 5: 779-88.
Cavazzana-Calvo M,, Hacein-Bey-Abina S, Fischer A. Déficits Immunitaires Primitifs et Thérapie Génique. Le Point 2005, Biotech Med. 2005; 32: 9-12.
Cavazzana-Calvo M,. Cellules souches et révision de la loi de bioéthique : les retards français. Les Cahiers du C.C.N.E. 2004; 41: 47-8.
Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. Thérapie génique chez des enfants atteints du déficit immunitaire combiné sévère lié à l’X : efficacité et complications. Médecine Sciences. 2004; 20: 115-7.
Cavazzana-Calvo M,, Lagresle C, André-Schmutz I, Hacein-Bey-Abina S. Moelle osseuse : réservoir de cellules souches réparatrices de différents tissus ? Ann Biol Clin. 2004; 62: 131-8.
Cavazzana-Calvo M,, Hacein-Bey-Abina S, Le Deist F, Fischer A. Ils vont bien… un an après. Médecine Sciences. 18: 797-8.
Cavazzana-Calvo M,André-Schmutz I, Lagresle C,Fischer A. Medical perspectives of adults and embryonic stem cells. C R Biol. 2002; 325: 1053-8.
Hamel Y, Andre-Schmutz I, Bonhomme D, Cavazzana-Calvo M. Immunothérapie adoptive antivirale et transplantation. Le courrier de la transplantation. 2001; 3: 118-9.
Cavazzana-Calvo M. La thérapie génique des déficits immunitaires congénitaux. Med Thérapeutique.2001; 7: 719-24.
Hacein-Bey S, Gross F, Nusbaum P, Yvon E, Fischer A, Cavazzana-Calvo M. Thérapie génique du deficit immunitaire combiné sévère lié à l’X. Pathol Biol. 2001; 49: 57-66..
Fischer A, Hacein-Bey S, Le Deist F, de Saint-Basile G, de Villartay JP, Cavazzana-Calvo M. Gene therapy of immunodeficiency disorders. Bull Acad Natl Med. 2000; 178: 13-20.
Fischer A, Hacein-Bey S, Le Deist F, Cavazzana-Calvo M. Traitement du déficit immunitaire combiné sévère lié à l’X par transfert ex-vivo du gène gamma c. Médecine Sciences. 2000; 16: 681-4.
Fischer A, Hacein-Bey-Abina S, Le Deist F, de Saint-Basile G, de Villartay JP, Cavazzana-Calvo M. Thérapie génique des déficits immunitaires. Bull Acad Natl Med. 2000; 184: 1417-28.
Cavazzana-Calvo M, Hacein-Bey-Abina S, de Saint-Basile G, Le Deist F, Fischer A. Thérapie génique des déficits immunitaires combinés sévères. Transfus Clin Biol. 2000; 7: 259-60.
Fischer A, de Saint-Basile G, Hacein-Bey S, Soudais C, Di Santo J, Cavazzana-Calvo M. Thérapie génique des déficits immunitaires: approche expérimentale et premiers résultats cliniques. Médecine Sciences. 1999; 15: 606-14.
Fischer A, de Saint-Basile G, Di Santo JP, Hacein-Bey S, Sharara L, Cavazzana-Calvo M. Thérapie génique pour déficits immunitaires héréditaires. C R Séances Soc Biol Fil. 1996; 190: 77-93.
Fischer A, de Saint-Basile G, Di Santo JP, Hacein-Bey S, Sharara L, Cavazzana-Calvo M. Thérapie génique des déficiences immunologiques héréditaires. Arch Pediatr. 1996; 3: 69s-76s.
Valteau-Couanet D, Cavazzana-Calvo M, Fischer A. Lymphocytes résiduels après dépletion spécifique. Etude fonctionnelle. Presse Med. 1992; 21:1939-40.
Gluckman E, Mazeron Mc, Keable H, Meletis J, Bombail D, Jolivet I, Huart JJ, Nebout T, Cavazzana M, de Castro H, Devergie A, Vilmer E, Perol Y. Prévention et traitement des infections à cytomégalovirus après greffe de moelle osseuse allogénique. Nouv Rev Fr Hematol. 1987; 29:17-21.
Gluckman E, Mazeron Mc, Keable H, Meletis J, Bombail D, Jolivet I, Huart JJ, Nebout T, Cavazzana M, de Castro H, Devergie A, Vilmer E, Perol Y. Nouvelle approche de traitement des infections à cytomégalovirus après greffe de moelle osseuse allogénique. Médecine et Maladies Infectieuses. 1987; 8/9:436-41.
Lemercier N, Varin F, Benbunan M, Cavazzana M, Miglierina R, Gluckman E, Reviron J. Techniques d’elimination des cellules T du greffon médullaireallogénique et méthodes d’evaluation des résultats. Actualités Hématologiques. Masson ed, 1986; 20 : 189-97.

Book Chapters

Cavazzana M. Overview of Immunodeficiency (Encyclopedie d’Immunologie). Encyclopedia of Immunobiology, 2nd edition, 2024
Cavazzana M, Diana JS. Thérapie génique. La drépanocytose de l’enfant et l’adolescent. Elsevier Masson, 2020.
Hacein-Bey Abina S, Fischer A.,Cavazzana M. Gene therapy for Severe Combined Immunodeficiencies (SCID). Advanced Textbook on Gene Transfer, gene Therapy and Genetic Pharmacology – 2^nd Edition. In World Scientific, 2020; 391-410.
Payen E, Colomb C, Negre O,Cavazzana M, Havein-Bey Abina S, Beuzard Y, Leboulch P. Gene therapy for β-hemoglobinopthies. Advanced Textbook on Gene Transfer, gene Therapy and Genetic Pharmacology – 2^nd Edition. In World Scientific, 2020; 441-452.
Cavazzana M,, Miccio A. Molecular based therapeutic approaches for b-hemoglobinopathies. Hematology Education: the education program for the annual congress of the European Hematology Association. Hematology Educational Book, Vienna June 2015; 103617.
Fischer A., Hacein-Bey Abina S.,Cavazzana M. How primary immunodeficiencies have made gene therapy a reality. Primary Immunodeficiency Disorders-Ebook- 327-339. 2014.
Cavazzana M.. The potential of genetically modified cell therapies.
Hacein-Bey-Abina S, Fischer A, Cavazzana-Calvo M. Applications: X-linked severe combined immunodeficiency and other hereditary diseases. In: Morishita R, Nakagami H (Eds) Gene Therapy: Technologies and Applications; E-Book, Future Science Group, 2012, chap. 5, pp.
Cavazzana-Calvo M.. Thérapie Génique. In « Les Cibles Nucléaires en Oncologie », AMGEN Oncologie and John Libbey Eurotext, 2012 ; pp.100-110.
Cavazzana-Calvo M., Hacein-Bey-Abina S, Fischer A. Therapy of primary immunodeficiencies: promises and culprits of gene therapy. In EHA Education Book Amsterdam April 2012.
Cavazzana-Calvo M., Leboulch P, Negre O, Payen E, Fischer A, Hacein-Bey-Abina S. Thérapie génique des maladies du système hématopoïétique. In Journées Parisiennes de Pédiatrie 2011, Nex & Com Medical Events, Boulogne Billancourt, 2011, pp. 143-151.
Cavazzana-Calvo M.. Les thérapeutiques des déficits immunitaires. In Biologie Géologie, APBG, Paris, 2011, 2 :129-41.
Cavazzana-Calvo M., Lagresle-Peyrou M, Rivière J, Calvo C, Hacein-Bey-Abina S. Thérapie génique. In: Romana S, Bonnefont JP, Cavazzana-Calvo M, Malan V, Jais JP (Eds) Méthodes d’Etude et d’Analyse du Génome ; Elsevier-Masson, Paris, 2011, chap.4, pp.87-102.
Cavazzana-Calvo M., Friedrich W, Fischer A. Hematopoietic stem cell transplantation for children with severe combined immunodeficiencies. In: Apperley J, Carreras E, Gluckman E, Gratwhol A, Masszi T (Eds), Haemopoietic Stem Cell Transplantation, ESH-EBMT Handbook, 2008, Chap. 40, p. 536-43.
André-Schmutz I, Cavazzana-Calvo M. Gene and cell therapy involving hematopoietic stem cell. In: Godin I & Cumano A (Eds), Hematopoïetic Stem Cell Development Landes Bioscience/Springer-Verlag, 2006, Chap. 12.
Cavazzana-Calvo M,Hacein-Bey-Abina S, Thrasher AJ, Leboulch P, Fischer A. Correction of genetic blood defects by gene transfer. In: Runge MS & Patterson C (Eds), Principles of Molecular Medicine, 2nd ed., Human Press Inc., Totowa, New Jersey, 2006, Chap. 88, p.854-61.
Steward CG, Cavazzana-Calvo M. Gene Therapy. In: Murphy M & Pamphilon D (eds), Practical Transfusion Medicine, 2^nd Ed, Blackwell Publishing Ltd, Oxford, 2005, Chap. 34, p.390-402.
Cavazzana-Calvo M,Le Deist F. Stem cell transplantation in children: Severe combined immunodeficiency. In: Apperley J, Carreras E, Gluckman E, Gratwohl A, Masszi T (Eds), Hematopoietic Stem Cell Transplantation, ESH/EBMT Handbook, 2004, Chap. 17.3, p.308-13.
Cavazzana-Calvo MThérapie cellulaire. In: Chouaib S & Bensussan A (eds), Immunologie des Cancers, Médecine-Sciences Flammarion, 2003, Chap.15, p.171-9.
Cavazzana-Calvo M, Hacein-Bey S, Gross F, Nussbaum P, Hue C, De Villartay JP, de Saint-Basile G, Le Deist F, Fischer A. Thérapie génique des déficits immunitaires héréditaires. In: Cohen-Haguenauer O (Ed), Thérapie Génique, Editions Tec et Doc, 2001, Chap. 24, p.369-82.
Cavazzana-Calvo M, Bagnis C, Mannoni P, Fischer A.Peripheral blood stem cell and gene therapy. In: Gorin NC (Ed), Clinical Haematology – Peripheral Stem Cells in Bone Marrow Transplantation, Bailliere Tindall, London, 1999, Chap. 10, p.129-38.
Fischer A, de Saitn-Basile G, Di Santo JP, de Villartay JP, Hivroz C, Cavazzana-Calvo M, Le Deist F. Severe combined immunodeficiencies.In: Weir DM (Ed), Weir’s Handbook of Experimental Immunology, 5^th ed., Blackwell Publishing Ltd, 1996, Chap. 180.
Fischer A, Cavazzana-Calvo M, Jabado N, Sarnacki S. Monoclonal antibodies to adhesion molecules in bone marrow and organ transplantation. In: CHATENOUD L (Ed), Monoclonal Antibodies in Transplantation, Springer-Verlag, Austin, Texas, 1995, Chap. 5, p.137-53.
Basso G, Foa R, Putti MC, Cavazzana-Calvo M, Testi AM, Capuzzo F, Cozzi M, Milanesi C, Consolini R, Galdiolo D, Cantoretti G, Zanesco L. Leucemies aiguës HLA-DR+ Tdt+: caractérisation immunologique et immunocytochimiqueIn: Bernard A, Boumsell L, Demeocq F (Eds), Biologie des Leucémies et Hématosarcomes, Editions Gaston Lachurie, Paris, 1983, p. 73-8.

Textbooks

Marina Cavazzana. “ Prévenir et soigner la drépanocytose”. Chapter 14, Unit 2 of the Belin Life Science book for 11^th grade, page 245.
Cavazzana-Calvo M, Debiais M. Les Biomédicaments. Collection “Que Sais-je”, AMGEN / Presses Universitaires de France, Paris, 2011, 1 volume, 126 pages.

Editing activities

Encyclopedia of Immunobiology, Second Edition, Section Editor- 2023-2024 – in charge ofSection 19: Immune deficiency
Encyclopedia of Immunology. Associate Editor in charge of the section on immunodeficiencies. 2015-2016
Cavazzana-Calvo M, André-Schmutz I. Cytotherapy 2005; 7. Special Focus issue on alloselective approaches to optimize immune reconstitution after allogeneic hematopoietic stem cell transplantation.

CONGRESS LIST

Invited conferences

International

2025

51st Annual Meeting of the EBMT: Chair Session title: ATMPs (30 March-2 April 2025) Florence Italy
2024
The 66th Ash Annual Meeting And Exposition, Moderator: Understanding Immediate Toxicities of Current Gene and Cell Therapy Tools – Scientific Workshops / Presenter: Gene Therapy for Artemis-SCID and Artemis-Leaky-SCID Patients: Preliminary Results of the French Artegene Phase I/II Clinical Trial – Oral and Poster Abstracts / Speaker Moderator: Introduction (Classical Hematology Perspective) – Scientific Workshops / Speaker: The Role of Inflammation in Sickle Cell Disease Pathology – Scientific Workshops (December 7-10, 2024) San Diego USA
4th International WAS Symposium, – Panelist/Session Title: Gene Therapy Panel: Gene Therapy, why is there variability in outcome and how can we address it? (November 18, 2024) Milan Italy
EUHA EUCCAT: the possibility of providing an academic alternative to the current commercial product and how EUCCAT could help in providing this alternative – Bifunctional GLOBE-AS3 lentiviral vector expressing a miRNA against HbS-Online- (November 11, 2024) Milan Italy
31^th ESGCT/SITGEC Collaborative Congress (October 22-25, 2024) Rome, Italy
31^st Public MMPU Research – Day MMPU Research Day and advisors meeting: Speaker- Gene Therapy for Sickle Cell Disease What Have We Learnt – Guest of MMPU Group Translational
RNA Biology (TRB)- EMBL Heidelberg, Imaging Center Lecture Hall (October 15, 2024) Germany
Inborn Errors Working Party Annual Conference – Presentation Title: Gene Therapy for SCD: Achievements and issues to solve (September 27-29, 2024) Helsinki, Finland
ISCT EUROPE meeting- Session Title: Autologous Gene Modified HSC Therapies (September 4-6, 2024) Sweden

2023

65^th ASH Annual Meeting and Exposition, (December 9-12, 2023) San Diego USA
Workshop on ATMPs Cell and gene therapy for inherited and acquired disease of the hematopoietic system- Invited speaker (November 30 2023) Potsdam Germany
SFGM-TC 2023 CO-II-2: Exagamglogene autotemcel for transfusion-dependent β-thalassemia and severe sickle cell disease -Invited speaker-(November 15-17, 2023)
The ESGCT, SFTCG and NVGCT annual congress (speaker).. Parallel session 10c Bleeding disorders/ Parallel session 6d: Hematopoietic diseases gene therapy- Chair over a session: Parallel 10c: PID gene therapy-(October 24-27, 2023) Brussels
Annual Meeting,, International Society for Cell and Gene Therapy (ISCT) – Video conference Internationnal: Plenary Speaker (August 16-18, 2023) In Perth, Western Australia (WA)
XXIII Wilsede Meeting On Moodern Trends In Human Leukemia And Cancer Title : Gene therapy for hematopoietic inherited disorders : 20 years of progress (June 24-27,2023) wilsede, Germany
ISCT 2023- Annual meeting of the international Society of Cellular Therapy , (May 8-June 3, 2023)

Invited panel list for two roundtables :

point of care manufacturing : disrupting and decentralizing autologous delivery

pediatric patients : how to mesure the risk/benefits ?

2022

EBMT:T cell reconstitution and clinical outcome after allogeneic Hematopoietic Tranplanatation
CDCM 6^th annual symposium: Gene therapy for inherited disease of the hematopoietic system between safety ed Efficacy
4^th Global Congress on sickle cell disease: Gene therapy for SCD(Paris 2022) between safety and Efficacy
Science Summit at UNGA: Gene therapy for SCD (September 16, 2022)
EHA, Vienne 2022: Lecture for the Jose Carreras award

2021

Inborn Errors Working Party annual conference, ARTEMIS GT- HTLP (human Tlymphocyte progenitor). September 25, 2021
Brazilian Hermatology and Hemotherapy Association (ABHH): Advanced therapies: Celles and Genes. Novel vectors to cure sickle cell disease – 6-7 august 2021
CTTC annual conference, Gene therapy for β hemoglobinopathies: lentiviral and Genome Editing Approaches- Juin 2021. Virtual conference.
Lezioni di Medicina Molecolare, Collegio Ghislieri, Universita di Pavia, “Terapia genica per le malattie ereditarie del sistema Ematopoietico”. April 2021. Virtual conference.
Seminar sessions, Instituto Gulbenkian de Ciência. “Gene Therapy in Genetic Disorders”. April 2021. Virtual conference.
ESH 2^nd Translational Research E-Conference: ‘Erythropoiesis Control and Ineffective Erythropoiesis: from bench to bedside’. “Gene Therapy in haemoglobinopathies vs. BMT”. March 2021. Virtual conference.

2020

European HematologyAssociation (EHA). “Gene therapy or genome editing: How to choose”. June 2020. Virtual congress.
European Society of Human Genetics (ESHG). “Gene therapy in genetic disorders”. June 2020. Virtual congress.
International Society of Cell and Gene Therapy (ISCT). “Clinical breakthroughs in hematopoietic stem cell-based gene therapy”. May 2020. E-virtual congress.
International Society of Cell and Gene Therapy (ISCT). “Gene therapy for globin disorders”. May 2020. Virtual congress.
Keystone Symposia – Emerging Cellular Therapies: Cancer and Beyond, joint with Engineering the Genome. “Gene Therapy for Immunodeficiencies”. February 2020. Banff, Canada.

2019

Stem Cell Transplantation in Childhood Congress. “Gene therapy and genome editing in 2018”. Septembre 2019. Roma, Italy.
International Congress on Research of Rare and Orphan Diseases (RE(ACT) Congress). “Gene Therapy for inherited diseases of the hematopoietic system”. May 2019. Toronto, Canada.
8^th Congress of the International BioIron Society (EMBL Conference). “Gene Therapy to cure Hemoglobinopathies”. May 2019. Heidelberg, Germany.
International Symposium Gene and Cell Therapy. “Gene Therapy for hematologic diseases”. April 2019. Zurich, Switzerland.
45^th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2019). “Lentiviral Gene Therapy”. March 2019. Frankfurt, Germany.
University of Chicago Center in Paris Colloquium, Global Health Colloquium focused on Doctor-Patient Relationship. “Gene Therapy for Hemoglobinopathies”. February 2019. Paris, France.
First edition of the DIM Thérapie Génique Symposium, “Gene Therapy for Rare Monogenic Diseases. Institute Imagine, February 2019 Paris, France.

2018

Brazilian Congress of Hematology, Hemotherapy and Cell Therapy (HEMO 2018). “Gene Therapy in Sickle Cell Disease”. November 2018. São Paulo, Brazil.
European Society for Immunodeficiencies (ESID). “Novelties in Gene Therapy: Further paving the way”. October 2018. Lisboa, Portugal.
European Society of Gene and Cell Therapy (ESGCT). “Gene Therapy of Hemoglobinopathies”. October 2018. Lausanne, Switzerland.
International Society for Cellular Therapy EU (ISCT EU). “Gene Therapy for Haematoglobinopathies: French Experience”. September 2018. Firenze, Italy.
Advances in Haematology in Africa Course. “Gene Therapy of SCD”. August 2018. Dar Es Salaam, Tanzania.
World Federation of Hemophilia. “What can we learn from gene therapy for other inherited blood disorders”. May 2018. Glasgow, United Kingdom.
Stanford University of Medicine. “Gene therapy for haemoglobinopathies: advances and challenges”. January 2018. Stanford, USA.
9^th international symposium on Haploidentical HSCT conference. Weizmann Institute of Science. January 2018. Rehovot, Israel.
51^st Miami Winter Symposium Stem Cells – Today’s Research Tomorrow’s Therapies. “Primary Immunodeficiencies Gene Therapy”. January 2018. Miami, USA.

2017

1^st International Symposium on Inner Ear Therapies. “Gene therapy: not only a matter of vector choice”. November 2017. Marrakesh, Morocco.
1^st International Working Group on Thalassemia. “Highlights on gene therapy in haemoglobinopathies”. September 2017. Palermo, Italy.
International AIDS Society Conference on HIV Science. “Gene therapy: challenges to face for patient’s benefit”. July 2017. Palais des Congrès, Paris, France.
Hellenic Society Gene Therapy and Regenerative Medicine. “Gene therapy”. May 2017. Athens, Greece.
British Society for Gene and cell Therapy (BSGCT). Scientific Breakthroughs session “Results and challenges of ex vivo gene therapy clinical trials”. April 2017. Cardiff, Wales, United Kingdom.
France-India Heathcare Summit 2017. “Rare diseases and gene therapy”. March 2017. Paris, France.
European Society for Blood and Marrow Transplantation (EBMT). “SCIDX1”. March 2017. Marseille, France.

2016

Indian Society of Hematology and Blood Transfusion (ISHBT). “Gene therapy for haemoglobin disorders – How we define success?”. November 2016. Jaipur, India.
10^th anniversary conference of the Academy for Sickle Cell and Thalassemia. “Gene Therapy for hemoglobinopathies”. October 2016. London, UK
XLP-WAS 2016 Symposium. “Gene therapy of WAS: There is room for further improvements”. September 2016. London, UK.
6^th International symposium on sickle cell disease in Central Africa. “Gene therapy for sickle cell disease”. May 2016. Kampala, Uganda.
Primary Immune Deficiency Treatment Consortium. Rapid turnaround antiviral T cell therapy (Gammacapture study) and Gene therapy. May 2016. Los Angeles, USA.
Institut de Génétique et de Biologie Moléculaire et Cellulaire. Gene therapy of hereditary diseases affecting the hematopoietic system. April 2016. Illkirch, France.
Global Iron and hematopoiesis summit. Recent advances in gene therapy for hemoglobinopathies. March 2016. Madrid, Spain.
Advances of gene therapy in monogenic diseases affecting the hematopoietic system workshop. “Hematopoietic stem cell gene therapy trials at the Necker Hospital”. February 2016. Madrid, Spain.
Weatherall Institute of Molecular Medicine. “Clinical application of gene therapy in hematology, where do we stand?” January 2016. University of Oxford, Oxford, UK.

2015

Tenth Cooley’s anemia symposium. Session Gene Therapy “Updates on LentiglobinÒ Gene Therapy Clinical Trials. October 2015. Chicago, USA.
50 years of scientific excellence in medicine at the Hannover Medical School. Talk session “Gene Therapy moves from simple rare primary diseases to more frequent and complex ones”.September 2015. Hannover, Germany.
EMBO Conference. Cell Therapy today: achievements, hope and hypes. Session Hematopoietic Cells “Gene therapy of hemoglobinopathies: clinical results and future developments”. September 2015. Manchester, UK.
Scientific Meeting of the Department of Molecular Medecine of the University of Padua. Session 3: Advanced cell and molecular therapies. “Molecular based therapeutic approaches in primary immunodeficiencies and AIDS”. May 2015. Padova, Italy.
British Society of Blood and Marrow Transplantation (BSBMT) Scientific Day. Gene editing for immune and haematological disorders: “Gene therapy for inherited disorders”. May 2015. London, UK.
European Society for Blood and Marrow Transplantation (EBMT). Working Party: Paediatrics Diseases.” Gene therapy clinical trials: recent progress”.March 2015. Istanbul, Turkey.

2014

16^th Biennial Meeting of the European Society for Immunodeficiencies (ESID). Chairman Parallel Session X: “Hematopoietic Stem Cells Transplantation and Gene Therapy”. October – November 2014. Prague, Czech Republic.
Annual meeting of the European Society of Gene and Cell Therapy (ESGCT). “Highlights of Clinical Progress”. October 2014. The Hague, The Netherlands.
19^th Congress of the European Hematology Association (EHA). “EHA Education Session”. June 2014. Milano, Italy.
20^th annual meeting of the International Society for Cellular Therapy (ISCT). Presidential plenary session:” Gene therapy with self inactivated retrovirus vector: a great step forwards the clinical front”. April 2014. Paris, France.
24^th Annual meeting of the society for virology. Plenary lecture “Gene therapy for inherited disorders: lessons for a broader use”. March 2014. Innsbrück, Austria.
Belgian Society of Hematology general annual meeting. “Gene therapy of hemoglobinopathies”. January 2014. Brussels, Belgium.

2013

TEDxLakeComo. “Gene therapy today”.November 2013. Como, Italy.
Scientific Conference. “Clinical results of gene therapy for blood diseases”. November2013.Luxembourg.
World Cord Blood Congress IV and Innovative Therapies for Sickle Cell Disease. October 2013. Monaco.
5^thEuropean Congress of Virology. “Using retroviruses for human genetic therapy: progresses and difficulties”. September 2013. Lyon, France.
Immune Deficiency Foundation National Conference. “Gene therapy for WAS, HSCT for WAS, N-plate to treat thrombocytopenia”. June 2013. Baltimore, USA.
International AIDS Society “Towards an HIV Cure” Think-Tank Meeting. “HIV Gene Therapy: Are we there yet?”. May 2013. Pasteur Institute, Paris, France.
Pasteur Institute International Meeting “30 Years of HIV Sciences: Imagine the future”. “Gene therapy approach for HIV-1 infection”. May 2013. Paris, France.
20^th Conference on Retroviruses and Opportunistic Infections (CROI). Plenary lecture:”Treatment with Gene-modified Hematopoietic Stem Cells May Definitively Abolish HIV-1 Infection”. March 2013. Atlanta, USA.
Miltenyi Biotec MACS Symposium: The new era of vaccination: the antigen specific adoptive T-cell therapy. “How to protect the recipient of an HLA partial incompatible HSCT from the infectious risk?”. February 2013. Florence, Italy.

2012

17^th Congress of the Asia-Pacific Blood and Marrow Transplantation group (APBMT). Plenary Lecture“Autologous gene modified HSC for benign hematological diseases: myth or reality?” October 2012. Hyderabad, India.
41^st Annual Meeting of the International Society for Experimental Hematology (ISEH). “Hematopoietic stem cell transplantation versus gene therapy for inherited disorders of the hematopoietic system”. August 2012. Amsterdam, The Netherlands.
International Symposium – The Future of Paediatric Immunology: From Cellular Pathology to Molecular Therapy. “Lessons from PID gene therapies”. July 2012. Zürich, Switzerland.
17^th Congress of the European Hematology Association (EHA). “Therapy of primary immunodeficiencies: promises and culprits of gene therapy”. June 2012. Amsterdam, The Netherlands.
Freiburg Symposium on “Immunodeficiency and Beyond”. “Immunodeficiencies: from the molecular diagnosis to innovative therapies”. March 2012. Freiburg, Germany.
75^th Anniversary of Albert Szent-Györgyi Nobel Prize Award. “Gene therapy for inherited diseases of the hematopoietic system: from bench to the bedside”. March 2012. Szeged, Hungary.
2^nd Congress of the Hellenic Society of Haematology – Oncology. “Gene therapy for primary immunodeficiencies and red blood cell diseases”. February 2012. Thessaloniki, Greece.

2011

3^rd World Cord Blood Congress. “Advances in adoptive immunotherapy to accelerate T-cell immune reconstitution”. October 2011. Roma, Italy.
Red Cell Disorders European Workshop. “Thalassemia: indication to HLA non-identical family member donor HSCT”. September 2011. Vienna, Austria.
36^th FEBS Congress, “Science & Society” Session on Genetic Diseases. “Gene therapy: acquired results, expected advances and obstacles”. June 2011. Torino, Italy.
16^th Congress of the European Hematology Association. “Gene therapy and Thalassemia”. June 2011. London, UK.
Memorial Sloan-Kettering Cancer Center Special Seminar. “How can we accelerate T-cellular immune reconstitution after incompatible hematopoietic stem cell transplantation?”. May 2011. New York, USA.
Life-Sciences Seminar of the Ecole Polytechnique Federale de Lausanne (EPFL). “Gene therapy for inherited diseases of the hematopoietic system”. April 2011. Lausanne, Switzerland.
Montreal Jean Pouliot Telethon Lectureship. “Gene therapy for hematopoietic stem cells inherited diseases: lessons from the past and future plans”. March 2011. McGill University Hospital for Children, Montreal, Canada.
Montreal Jean Pouliot Telethon Lectureship. “Comparison of outcomes after mismatched related stem cell and unrelated cord blood transplantation in children with severe T-cell deficiencies”. March 2011. Saint-Justine Hospital, Montreal, Canada.
Montreal Jean Pouliot Telethon Lectureship. “Adenylate kinase 2 deficiency”. March 2011. Montreal, Canada.
London – Padua – Paris Labs Network, 1^st International Workshop on Regenerative Medicine in Pediatrics. “Gene therapy: which improvements are expected in short and long term?”. February 2011. Padua, Italy.
University of Toronto Department of Immunology’s Easton Seminars. “Gene therapy for inherited disorders: ongoing clinical trials and future developments”. January 2011. Toronto, Canada.
University of Toronto Panel Discussions in Human Immunology. “Hereditary immune deficiencies: lessons for a better understanding of the physiology of immunological development and immune therapeutics approach”. January 2011. Toronto, Canada.

2010

Boston Children’s Hospital Dobuler Grand Round Lecture. “Gene therapy for inherited disorders of the haematopoietic system: where do we stand 10 years after the SCID-X1 trial”. December 2010. Boston, USA.
1^st International Fanconi Anaemia Working Group Meeting. “SCID, beta-thalassemia and ALD gene therapy trials”. November 2010. London, UK.
ESH Eurocord-Ed, Eurocord, Netcord World Cord Blood Congress. “Gene therapy for haemoglobinopathies”. November 2010. Marseille, France.
ESH Eurocord-Ed, Eurocord, Netcord World Cord Blood Congress. “Comparison of haploidentical and cord blood HSCT in children with immune deficiencies”. November 2010. Marseille, France.
Fred Hutchinson Cancer Research Center (FHCRC) Clinical Research Division Seminar. “Gene therapy clinical trials ongoing at Necker Enfants Malades Hospital”. April 2010. Seattle, USA.
BMT Tandem Meetings. “Haploidentical bone marrow transplantation for primary immunodeficiencies: obstacles and anticipated progress”. February 2010. Orlando, USA.

2009

International Symposium on Immunotherapy and Immunodeficiency. “Severe combined immunodeficiency: from molecular immunology to therapy”. November 2009. Heidelberg, Germany.
Fondazione Internazionale Menarini Meeting: Focus on Cell Therapy – Transplantation and Tissue Repair. “Cell and gene therapy for primary immunodeficiencies and other hereditary diseases”. October 2009. Bergamo, Italy.
International Union of Immunological Societies (IUIS) Expert Meeting on Primary Immunodeficiencies. “Adenlyate kinase 2 deficiency”. June 2009. Dublin, Ireland.

2008

8^th International Conference on “The Biology of Stem Cells”. “Innovative cell therapy protocols for inherited diseases”. November 2008. Institute of Integrative Biology (Université Pierre et Marie Curie), Paris, France.
12^th Meeting of the Portuguese Society of Human Genetics. “Clinical results and biological achievement of gene therapy protocols addressed to inherited diseases”. November 2008. Lisbon, Portugal.
Annual Congress of the Japanese Society of Pediatric Oncology and Haematology. “Gene therapy for primary immunodeficiencies”. November 2008. Tokyo, Japan.
2^nd Meeting of the Japanese Society of Immunodeficiency. “Human lymphoid progenitor cells and its disease”. November 2008. Tokyo, Japan.
Kyoto University Hospital, Translational Research Unit Seminar. “Gene and cell therapy for pediatric diseases”. November 2008. Kyoto, Japan.
Ministry of Academic Teaching and Research International Conference on “Science in Society: Dialogues and Scientific Responsibility”. “Stem Cells – from basic research to applications: the society expectations”. November 2008. Paris, France.
13^th Meeting of the European Society for Immunodeficiencies (ESID). “What to do when an HLA identical donor is not available. Cord blood versus haplo: focus on SCID”. October 2008. Hertogenboshc, The Netherlands.
13^th Meeting of the European Society for Immunodeficiencies (ESID). “Human adenylate kinase 2 deficiency causes a very profound haematopoietic defect associated with sensorineural deafness: reticular dysgenesis”. October 2008. Hertogenboshc, The Netherlands.

2007

Max Planck Institute for Infection Biology Panel Discussion on Ethical Aspects of Gene Therapy. “Gene therapy for primay immunodeficiencies”. December 2007. Berlin, Germany.
Padua University Department of Pediatrics Seminar on Stem Cell Research. “Update on the future of stem cell research”. November 2007. Padua, Italy.
Padua University Department of Pediatrics Seminar on Stem Cell Research. “Gene therapy of DICS-X1”. November 2007. Padua, Italy.
European Science Foundation EuroSTELLS Workshop “Challenges in Stem Cell Differentiation and Transplantation”. “Bone Marrow Transplantation in Primary Immunodeficiencies”. September-October 2007. Milano, Italy.
4^th Conference on Stem Cell Gene Therapy, University of Washington / Hellenic Society of Haematology. “Gene therapy targeting hematopoietic stem cells: from the bench to the bedside”. September 2007. Chalkidiki / Thessaloniki, Greece.
10^th Annual Meeting of the American Society of Gene Therapy. “Ethics of patient selection for severe immunodeficiencies”. May-June 2007. Washington, USA.
Seminar on Gene Therapy. May 2007.Department of Microbiology, Unviersity of Pennsylvania School of Medicine, Philadelphia, USA.
Multidisciplinary Session of the Triennale di Milano “From molecules to population studies: a travel in the world of medicine and healthcare”. “How can we modify the behaviour of a cell through genetic engineering?”. May 2007. Milano, Italy.
Seminar on Gene Therapy for Immunodeficiencies. “Gene Therapy of SCID-X1 and other immunodeficiencies”. January 2007. Modena, Italy.

2006

12^thMeeting of the European Society for Immunodeficiencies. Workshop on Gene Therapy. October 2006. Budapest, Hungary.
1^st Joint Meeting of European National Societies of Immunology, 16^th European Congress of Immunoloy. “Introduction to Gene Therapy”. September 2006. Paris, France.
France-Israel Meeting on Stem Cells & Regenerative Medicine. “Human hematopoietic stem cells: biological advances and clinical applications”. September 2006. Haifa, Israel.
11^th International Congress of Human Genetics Medicine. “Gene therapy for severe combined immunodeficiency X1”. September 2006. Haifa, Israel.
11^th International Congress of Human Genetics. “Gene therapy for severe combined immunodeficiency X1”. August 2006. Brisbane, Australia.
31^st Federation of European Biochemical Societies Congress, Molecules in Health Disease. “Gene Therapy for Primary Immunodeficiencies: Progess and Obstacles”. June 2006. Istanbul, Turkey.
ECM International Forum Nuove Frontiere in Medicina. “Il Trapianto di Cellule Staminali Ematopoietiche”. June 2006. Padova, Italy.
European Commission Research Workshop on Gene Therapy. “Gene Therapy of Hematopoietic Diseases”. June 2006. Brussels, Belgium.
Cold Spring Harbor Laboratory Meeting on Retroviruses. “Primary immunodeficiency and genetics”. June 2006. Columbia University, NY, USA.
11^th Congress of the European Association Hematology. Scientific and Education Committee of the 11th Congress of the European Hematology Association. March 2006. Amsterdam, The Netherlands.
International workshop on « Options for the management of ADA-SCID”. March 2006. Hambourg, Germany.
32^nd Annual Meeting of the European Group for Blood and Marrow Transplantation. March 2006. Hambourg, Germany.
International Stem Cell Forum 2006. “Stem Cell Research in France: progresses and objectives from basic to translational research”. January 2006. Paris, France.

2005

Seminar “Gene Therapy for Primary Immunodeficiencies Progress and Obstacles”. November 2005. Turin, Italy.
FAID (French-American Innovation Day) 2005 “Genes for therapy?” October 2005. Boston. USA
Conference “Cell and Gene Therapy”. October 2005. Barcelona, Spain.
International Symposium. “Primary immunodeficiencies in children”. September 2005. Zurich, Swizerland.
61^st National Congress of the Italian Pediatric Society. “Terapia Genetica de Malattie Ereditarie”. September 2005. Montecatini-Terme, Italy.
Riken Kobe Institute Center for Developmental biology Trilateral Stem Cell Meeting. May 2005. Osaka. Japan
Seminar “Infection and Immunity”. April 2005. London. UK
European Group for Blood and Marrow Transplantation (EBMT) Euroconference on Stem Cell Research. April 2005. Cascais. Portugal
Georg Speyer Haus Symposium. “Gene therapy with gene modified adults stem cells”.March 2005. Frankfurt. Germany
Stem Cell Gene Therapy (SCGT). “Gene therapy of SCID”. January 2005. Pamplona. Spain

2004

8^th SIES Congress. “Terapia Genica”. September 2004. Pavie. Italy
Gene Therapy for Wiskott-Aldrich-Syndrome meeting. September 2004. Washington. USA
Grand Rounds, Children’s Hospital and Regional Medical Center/University of Washington. “Advances in the treatment of Primary Immunodeficiencies”. August 2004. Seattle. USA
Eurocancer 2004. “Gene Therapy: Bilogical and Clinical Lessons”. July 2004. Paris. France
Canadian Blood and Marrow Transplant Group. “Transplantation and Gene Therapy for Immune Disorders”. June 2004. London. Uk / Ontario. Canada
EAACI Congress. “Therapeutic modalities of immunodeficiencies”. June 2004. Amsterdam. The Netherlands
ISCT 10^th Annual Meeting. “Gene Therapy for Primary Immunodeficiencies: clinical results and future developments”. May 2004. Dublin. Ireland
European Interdisciplinary Academy of the Sciences. “Skills and Hopes of Gene Therapy”. March 2004. Paris. France
Symposium 150^th anniversary Paul Ehrlich Institut. “Clinical and Biological Lessons from the Gene Therapy Trial for SCID-X1 Patients”. March 2004. Langen. Germany
EUROLENTI/INHERINET meeting. “Current State of the RAG project”. January 2004. Barcelona. Spain

2003

11^th Congress of the European Society of Gene Therapy (ESGT). “SCID-X1 Gene Therapy: trials and tribulations “. November 2003. Edinburgh. Scotland
25 Jahre Department Forschung. “Gene Therapy for Human Severe Combined Immunodeficiencies”. September 2003. Basel. Switzerland
7^th European Biotechnology Crossroads. “Biotherapies, from promise to practice: Gene Therapy”. September 2003. Nantes. France
Miltenyi Biotec Ltd. Immunotherapy Meeting. “Gene therapy for X-linked SCID”. February 2003. Bristol. UK
International Symposium: Standardization and quality control of cell and gene therapy products. “Gene therapy of SCID-X1: practical experience in product development”. February 2003. Strasbourg. France
FDA meeting. “Genotoxicity in the SCID-X1 clinical trial”. February 2003. Washington, USA.

2002

44^th Annual Meeting of the American Society of Hematology (ASH). Speaker and chairwoman. December 2002. Philadelphia. USA
9^th Day of Clinical Immunology. November 2002. Geneva. Switzerland
Annual Scientific Meeting. September 2002. Utrecht. The Netherlands
Seminar at Dibit. June 2002. Milan. Italy
Clinical application of stem cells: International Conference “Stem cells and cell therapy”. May 2002. Academy of Sciences. Paris. France
Human Genome: XXIst Century. Impact in Pharmaceutical Sciences. May 2002. Lisbon. Portugal

2001

International symposium on transplantation and gene therapy. October 2001. Idar-Oberstein. Germany.
Gene therapy of human inherited immunodeficiencies: The Arosenius Symposium. September 2001. Malmö. Sweden.
30^th Annual Meeting of the International Society for Experimental Hematology. August 2001. Tokyo. Japan.
6^th Meeting of the European Haematology Association. June 2001. Frankfurt. Germany.

1997 – 2000

Annual meeting of the Japanese Society of Hematology. “Gene therapy of human SCID conditions”. December 2000. Kyoto. Japan.
Workshop on advanced cellular therapy in the child’s best interest. September 2000. Pavia. Italy
8^th Meeting of the European Society of Gene Therapy (ESGT). October 2000. Stockholm. Sweden
Cold Spring Harbor Laboratories Meeting. “Gene therapy for X-linked SCID”. September 2000. Cold Spring Harbor. USA
Blood and Marrow Transplant Registry. March 2000. Anaheim. USA.
41^st Annual meeting of the American Society of Hematology (ASH). Plenary session: American Society of Hematology award for best research. December 1999. New Orleans. USA
European School of Haematology. “Oncogenesis of hematopoietic development”. February 1999. Sesimbra. Portugal
Workshop on haploidentical hematopoietic transplantation. January 1999. Eilat. Israel
Miltenyi Satellite Symposium ASH. Positive Selection of CD34 cells. December 1998. Miami. USA
11^th International Symposium on Molecular Biology of Hematopoiesis and treatment of myeloproliferative diseases. June 1998. Bormio. Italy
Symposium. “The developing immune system after stem cell transplantation”. February 1998. Ulm. Germany
10^th symposium on Molecular Biology of Hematopoiesis. July 1997. Hamburg. Germany
The European Society of Human Genetics. May 1997. Genoa. Italy
Baxter Satellite Symposium, European Society for Blood and Marrow Transplantation (EBMT). March 1997. Aix-les-Bains. France

National

2025

7th European CAR T-cell Meeting – Speaker: Gene therapy for hemoglobinopathies(February 6-8, 2025) Strasbourg

2024

SFGM-TC: Thérapie génique dans les hémoglobinopathies / CO-III-1 – Efficacy and safety of a single dose of exagamglogene autotemcel for transfusion-dependent beta-thalassemia and severe sickle cell disease -(November 20-22 2024) Toulouse
Presentation at the AP-HP Headquarters (St-Antoine site): Genetically modified stem cell transplant: a new therapy to cure thalassemia and sickle cell disease (November 05 2024)
Round Table “Living with sickle cell disease” the long-term success of gene therapy – Imagine Institute (october 17 2024)
10^thCongress of the French Society of Predictive and Personalized Medicine – Speaker: First authorized CAS9 treatment: Desktop publishing in the treatment of hemoglobin diseases (October 9-10 2024) Paris
OrphanDev and PedStart – MTI Day: Gene therapy for hematopoietic diseases: obstacles to overcome to promote their use (October 3, 2024) Orléans
Scientific Day of CRC: Gene therapy for hematopoietic diseases: challenges to solve, Keynote Lecture on Gene Therapy – (September, 19 2024)
Session of foreign members of the division of biological and pharmaceutical sciences, Communication “Innovations in gene therapy” National Academy of Medicine (March 27, 2024)
OECD High-Level Policy Forum on the Future of People-Centred Health Innovation (Speaker), Session 3 – Genomic and genetic medicine: What are the implications for health systems and care delivery? (January 22, 2024)

6th FSSCR Congress: Gene therapy of Sickle cell Disease: where we stand- Closing keynote speaker (January 22 to 24, 2024) Paris

2023

Conference M2 Stem Cell Biology Sorbonne University: Gene therapy of genetic disease of the hematopoietic system (September 27, 2023)
Live Webconference – Gene Therapy in Rare Diseases – The first series of multidisciplinary discussions, including experts from the major specialties involved in gene therapies, aimed at an audience of non-expert doctors, who may be faced with the identification, referral and/or monitoring of affected patients. (26 septembre 2023)

Round table:

Patient access to gene therapy in France through clinical trials or the possibility of early access to these innovative drugs

The organization of gene therapy in France through rare disease programs

Therapeutic decision-making for these treatments

https://rarealecoute.com/webinaires-therapie-genique/

2009 – 2021

7th Congress “Genomic Medicine»: First patients treated by genome editing in hemoglobinopathies. (September 30, 2021)
SFH Annual Congress, September 9-11, 2021
MCGRE Health Sector – National Day: Side effects of gene therapy in the treatment of hemoglobinopathies – June 25, 2021
7th National Day of Rare Immuno-Hematological Diseases, “Gene Therapies”. June 2021. Paris, France.
DIUHP Session Déficits Immunitaires Primitifs, « Gene Therapy for Primary Immunodeficiencies. April 2021 Paris, France.
10th Conference on Human and Medical Genetics. “Progress and Perspectives in Gene Therapies for Hemoglobinopathies.” January 2020. Tours, France.
Plenary Meeting of the HCB (Haut Conseil des Biothechnologies). “Gene therapy: what future for patients? June 2019. Paris, France.
USPC-NUS. “Gene therapy for inherited disease of the hematopoietic system: advances and challenges”. May 2018. Paris, France.
30th Educational Seminar. “Gene Therapy for Hematological Diseases”.May 2018. Arles, France.
17th Congress of the French Society of Bone Marrow Transplantation and Cell Therapy (SFGM-TC). “Gene therapy treatments”. November 2017. Nice, France.
National Academy of Pharmacy, Academic Session “Therapeutic Updates”. “Sickle Cell Disease”. November 2017. Paris, France.
S³Odéon Sciences Santé Société. “Sickle Cell Disease, AIDS: Our Friends the Viruses.” October 2017. Paris, France.
HEC Paris – Gene Therapy: From Rare Diseases to Oncology, What Advances? What Markets? “The Innovative Therapies Factory”. October 2017. Paris, France.
Paris Pediatrics Days. “CRISPR-Cas9, revolutions and perspectives in pediatrics”. October 2017. Paris, France.
Association Francophone de Génétique Clinique. “Gene therapy advances». June 2017. Paris, France.
5th Biomedicine Agency Days. “Evolution of medical practices at the gene level”. May 2017. Paris, France.
PharmaCité – Pharmaceutical Companies. “The Revolutionary Model of Rare Diseases”. December 2016. Paris, France.
Internal Seminar of the Imagine Institute. “Gene therapy clinical trials: advances and challenges.” December 2016. Paris, France.
Paris Pediatric Days. “Sickle Cell Disease in 2016: Health Disaster or Therapeutic Revolution in Sight.” October 2016. Paris, France.
Medical Innovation and Future Health Forum. “Cost/Benefit Assessment of Gene Therapy.” June 2016. Paris, France.
Institut Pasteur Translational Research Day. “Understanding and Modifying the Human Genome: Hopes and Ethical Implications.” April 2016. Paris, France.
National worshop on innovation in health. Organized by the French Ministry of Social Affaires, Health and Women’s rights. Debate and Conference. January 23-24, 2016. Paris, France.
Scientific workshop of the Francophone Society of Histocompatibility and Immunogenetics (SFXI). “Gene therapy of immune deficiencies”. January 20-21, 2016. Paris, France.
2^ndEuropean MD-PhD Conference, Association Médecine Pharmacie Sciences. “Using gene therapy for the hereditary diseases of the hematopoïetic system”. July 2013. Paris, France.
30^th JC Dreyfus Scientific Day.“Gene therapy for inherited blood disorders”. October 2013, Paris, France.
Annual Meeting of the Biomedicine Agency. “ISSCR guidelines on clinical translation applied to HSCT future applications”. December 2009. Paris, France.
Conference: “Gene therapy in severe combined immune deficiencies”. June 2002. Toulouse, France.

Oral presentations at International consortium meetings

European Society for Blood and Marrow Transplantation (EBMT) – Inborn Errors Working Party Meeting (IEWP) Annual Meeting. “Preclinical studies on CD4 gene therapy in IPEX”. Octobre 2019. London, UK.
NET4CGD Final Consortium Meeting. Clinical Trials Session “Presentation of the French patients and treatment”. April 2018. Evry, France.
3^rd Annual Meeting NET4CGD. April 2016. London, UK.
European CELL-PID Final Meeting. 1) “Update on WAS gene therapy”; 2) “Efficacy with clinical batch for Artemis”. March 2016. Franciacorta, Italy.
Annual Eurofancolen Meeting. February 2016. Madrid, Spain.
2^ndAnnual Meeting NET4CGD. March 2015. Frankfurt, Germany.
European CELL-PID Annual Meeting. 1) “Update on WAS gene therapy”; 2) “Update on SCID-XI gene therapy”; 3) “Gene modified hematopoietic stem/progenitor cells: therapy for combined immunodeficiencies (Artemis, Rag, ADA)”. February 2015. Stresa, Italy.
Eurofancolen Meeting: 1) “Assessment of the safety and efficacy of prerixafor plus G-SCF-mediated mobilization of CD34 cells”; 2) “Assessment of the safery and efficacy of the infusion of gene-corrected CD34 cells in FA-A patients”. January 2015. Madrid, Spain.
European Society for Blood and Marrow Transplantation (EBMT) – European Society for Imunodeficencies (ESID) – Inborn Errors Working Party Meeting (IEWP) Annual Meeting. “Gene Therapy”. October 2014. Munich, Germany.
8^th Annual Advanced Conference on Sickle Cell and Thalassaemia. “Gene therapy for
haemoglobinopathies: limitations, expectations and possibilities”. October 2014. London, UK.
Eurofancolen meeting. “Assessment of the safety and efficacy of the infusion of gene-corrected CD34⁺ cells in FA-A patients.” March 2014. Madrid, Spain.
European NET4CGD 1^st Annual Meeting. “Study in CGD”. February 2014. Stresa, Italy. European CELL-PID Annual meeting. 1) “Therapy for combined immunodeficiencies (Artemis, Rag, ADA); 2) “Implementation of novel clinical trials”; 3) “Phase I/II trial of SIN gammaretroviral gene therapy for SCID-X1”. February 2014. Stresa, Italy.
European Society for Blood and Marrow Transplantation (EBMT) Inborn Errors Working Party Meeting (IEWP). ” 15 years after 1^st SCT in SCID: back to the future”. September 2013. Leiden, The Netherlands.
30^thAnnual Meeting of Pediatric Immunology Working Group. “Gene therapy for T-cell deficiency syndromes”. September 2013. Düsseldorf, Germany.
Transatlantic Gene Therapy Consortium Meeting. “WAS Europe and SCID-X1 Clinical Trials”. October 2012. Evry, France.
6^th Stem Cell Clonality and Genome Stability Retreat. “Update of gene therapy WAS and thalassemia clinical trials in Necker hospital”. May 2012. Philadelphia, USA.
European Society for Blood and Marrow Transplantation (EBMT) Inborn Errors Working Party Meeting (IEWP). “Gene therapy for inherited blood disorders – future prospects”. April 2012. Geneva, Switzerland.
European CELL-PID Annual Governing Board Meeting. 1) “Gene modified hematopoietic stem/progenitor cells: therapy for combined immunodeficiencies (Artemis, Rag, ADA)”; 2) “The French cohort of CGD patients”; 3) “Gene transfer for Munc13.4 deficiency and IPEX studies”; 4) Update on WAS gene therapy”. January 2012. Stresa, Italy.
European CELL-PID Kick-off meeting. 1) “Lentiviral gene therapy for Artemis deficiency”; 2) Update on ongoing WASP clinical trial”. January 2011. Leukerbad, Switzerland.
European CELL-PID and PERSIST Joint meeting. “Update on ongoing SCID-X1 clinical trial”. January 2011. Leukerbad, Switzerland.
European Society for Blood and Marrow Transplantation (EBMT) Inborn Errors Working Party Meeting (IEWP). “Myelodysplastic syndromes after HSCT for reticular dysgenesis”. November 2010. Venice, Italy,
European Society for Blood and Marrow Transplantation (EBMT) Inborn Errors Working Party Meeting (IEWP). “Rag / Artemis gene therapy”. November 2010. Venice, Italy.
5^th Stem Cell Clonality & Genotoxicity Retreat. “Murine Arf-SCID models: their role in predicting gene therapy safety”. November 2009. Hannover, Germany.
European Society for Blood and Marrow Transplantation (EBMT) Inborn Errors Working Party Meeting (IEWP). “Gene therapy: update on recent lentiviral studies”. September 2009. Cambridge, UK.
European Society for Blood and Marrow Transplantation (EBMT) Working Party Inborn Errors Meeting (IEWP). “Gene therapy for SCID-X1”. September 2008. Sirmione, Italy.
4^th Stem Cell Clonality & Genotoxicity Retreat. “Arf-/- Rag1-/- double K.O mice: new model for evaluation of gene transfer risk for SCID”. June 2008. Boston, USA.
European Society for Blood and Marrow Transplantation (EBMT) Working Party Inborn Errors Meeting (IEWP). March-April 2008. Florence, Italy.
Consert European Project Principal Investigators’ Meeting. “Gene therapy: clinical trials update”. January 2008. Leukerbad, Switzerland.
European Society for Blood and Marrow Transplantation (EBMT) Working Party Inborn Errors Meeting (IEWP) on “Strategies in Stem Cell Transplantation for PID”, “Experience of cord stem cell transplantation in SCID”, October 2007, Arta de Mallorca, Spain
European Society for Blood and Marrow Transplantation (EBMT) Working Party Inborn Errors Meeting (IEWP). March 2007. Lyon, France.
European Society for Blood and Marrow Transplantation (EBMT) Board Retreat. October 2005. Francfort, Germany.
8^th Annual Congress of American Society of Gene Therapy and Stem Cell Clonality and Genotoxicity Retreat 2005. June 2005. St Louis, USA.
European Society for Blood and Marrow Transplantation (EBMT) Working Party Inborn Errors Meeting (IEWP). November 2004. Ulm, Germany.
Stem Cell Clonality and Genotoxicity Retreat. “Insertional gene activation in the gene therapy of human SCID “. December 2003. San Diego, USA.
European Society for Blood and Marrow Transplantation (EBMT) Working Party Inborn Errors Meeting (IEWP). “Alloreactive depletion in HLA partially incompatible setting”. October 2000. Perugia, Italy.

Oral presentations to International congresses selected on submitted abstract

Société Française d’Hématologie (SFH) “Exagamglogene autotemcel pour la β-thalassémie dépendante des transfusions et la drépanocytose sévère “. March 2024
ASH 2024 (San Diego, CA, Dec 7-10 “Betibeglogene Autotemcel (Beti-cel) Gene Addition Therapy Results in Durable Hemoglobin A (HbA) Production With up to 10 Years of Follow-Up in Participants With Transfusion-Dependent β-Thalassemia”
ASPHO 2024 “Efficacy and Safety of Betibeglogene Autotemcel in Pediatric Patients with Transfusion-Dependent β-Thalassemia”
65th Annual Meeting and Exposition (ASH) Improvement in Iron Burden in Patients with Transfusion-Dependent β-Thalassemia (TDT) Treated with Betibeglogene Autotemcel (Beti-cel) Gene Therapy: Up to 9 Years of Follow-up. December 2023, San Diego, USA.
65th Annual Meeting and Exposition (ASH) Sustained Efficacy, Safety, and Improved Quality of Life in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel). December 2023, San Diego, USA.
59^th Annual Meeting of the American Society of Hematology (ASH). Longer Term Follow-up on the First Patients with Severe Hemoglobinopathies Treated with Lentiglobin Gene Therapy. December 2017, Atlanta, USA.
58^th Annual Meeting of the American Society of Hematology (ASH). “Gene therapy studies in hemoglobinopathies: Successes and challenges”. December 2016. San Diego, USA.
Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) and International Society for Stem Cell Research (ISSCR). “Gene therapy of inherited disease: advances and challenges” October 2016. Florence, Italy.
17^th Biennal Meeting of the European Society for Immunodeficiencies (ESID). “Gene therapy clinical results”. September 2016. Barcelona, Spain.
19^th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). “Clinical outcomes of gene therapy with BB305 lentiviral vector for sickle cell disease and thalassemia”. May 2016. Washington, USA.
French Society of Hematology (SFH). Gene therapy for sickle cell anemia. April 2016. Paris, France.
Annual Congress of the French Society of Cell and Gene Therapy (SFTCG). Thérapie génique des hémoglobinopathies. March 2016. Marseille, France.
57^th Annual Meeting of the American Society of Hematology (ASH). “Outcomes of gene therapy for B-Thalassemia major and severe sickle disease via transplantation of autologous hematopoietic stem cells transduced ex vivo with a lentiviral b^A‑T87Q-globin vector”. December 2015. Orlando, USA.
9^th Annual Sickle Cell and Thalassaemia Advanced Conference. Talk session “Gene therapy in sickle cell disease and thalassemia – preclinical and clinical studies to date”. October 2015. London, UK.
39^th European Society for Blood and Marrow Transplantation (EBMT) Annual Meeting. “Allogeneic transplantation versus gene therapy: a difficult choice”. April 2013. London, UK.
3^rd International Fanconi Anemia Gene Therapy Working Group. “Gene therapy trials in Paris”. Octobre 2012. Versailles, France.
15^th Biennal Meeting of the European Society for Immunodeficiencies (ESID). “Gene therapy for Artemis deficiency”. October 2012. Florence, Italy.
53^rd Annual Meeting of the American Society of Hematology (ASH). “HSC Transplantation for Hemoglobinopathies: Allogeneic or Autologous Gene-Modified HSC?” December 2011. San Diego, USA.
14^thAnnual Meeting of the American Society of Gene & Cell Therapy (ASGCT). “Gene therapy for haematopoietic diseases: new data on ongoing clinical trials”. May 2011. Seattle, USA.
Pasteur Institute EMBO Conference on “Advances in stem cell research: development, regeneration and disease”. “Gene and cell therapy in hematopoietic system in human”. April 2011. Paris, France.
37^th Meeting of the European Society for Blood and Marrow Transplantation (EBMT). “Gene therapy for other haematopoietic disorders and future developments”. April 2011, Paris. France.
37^th Meeting of the European Society for Blood and Marrow Transplantation (EBMT), Miltenyi Corporate Satellite Symposium “Cellular Therapy: State of the art and future perspectives”. “How can we improve immunological reconstitution after hematopoietic stem cell transplantation”. April 2011. Paris, France.
European Society for Blood and Marrow Transplantation (EBMT) – Sickle Cell Disease Session: Gene Therapy for SCD. March 2011. Istanbul, Turkey.
17^th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). “From bench to bedside: ten years survey of a gene therapy clinical trial”. November 2009. Hannover, Germany.
11^th Annual Meeting of the American Society of Gene Therapy (ASGT). Symposium on Gene Therapy for Genetic Diseases: Update on Clinical Gene Therapy for Genetic Diseases. “Gene therapy SCID-X1 clinical trial: where do we stand?”. May-June 2008. Boston, USA.
15^th Annual Congress of the European Society of Gene & Cell Therapy. “Gene therapy for SCID-X1”. October 2007. Rotterdam, The Netherlands.
5^th Workshop on Haploidentical Stem Cell Transplantation. “Haploidentical bone marrow transplantation: how to speed up the immunological reconstitution? Current developments and obstacles to solve”. October 2007. Catania, Italy.
30^th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT). “Gene Therapy for Immunodeficiencies”. March 2004. Barcelona, Spain.
47^th Annual Meeting of the American Society of Hematology (ASH). “Gene therapy for severe combined immunodeficiency X1”. December 2005. Atlanta, USA.
46^th Meeting of the American Society of Hematology (ASH). December 2004. San Diego, USA.
45^th Annual Meeting of the American Society of Hematology (ASH). “Correction of SCID-X1 by gene transfer: current state and future plans”. December 2003. San Diego, USA.
6^th Annual Meeting of the American Society of Gene Therapy (ASGT). “Haploidentical Hematopoietic Stem Cell Transplantation Versus Gene Therapy for X-SCID Patients: Current State”. “SCID-X1 Clinical Trial: Current State”. Presentation of Top Three Abstracts. June 2003. Washington, USA.
43^rd Annual Meeting of the American Society of Hematology (ASH). December 2001. Orlando, USA
42^nd Annual Meeting of the American Society of Hematology (ASH). “Gene Therapy of human SCID-X1-disease”. December 2000. San Francisco. USA

Abstract selected for oral presentations and posters (congresses)

Amanda M. Li, Haydar Frangoul, Franco Locatelli, Akshay Sharma, Monica Bhatia, Markus Mapara, Lyndsay Molinari, Donna Wall, Laurence Dedeken, Robert Liem, Ami Shah, Paul Telfer, Selim Corbacioglu, Damiano Rondelli, Marina Cavazzana, Roland Meisel, Michael Eckrich, Stephan Lobitz, Mariane De Montalembert, Martin Steinberg, Mark Walters, Laura Bower, Suzan Imren, Chris Simard, Fengjuan Xuan, Weiyu Zhou, Phuong Khanh Morrow, William Hobbs, Stephan Grupp “Durable Clinical Benefits in Severe Sickle Cell Disease With Exagamglogene Autotemcel” – the Cell Therapy Transplant Canada Annual conference, held May 10-21-2025 in Québec City.
Mark C. Walters MD, Alexis A. Thompson MD, MPH , Timothy S. Olson MD, PhD , John B. Porter MD , Jennifer Schneiderman MD, MS , Suradej Hongeng MD , Andreas E. Kulozik MD, PhD , Marina Cavazzana MD, PhD, Martin G. Sauer MD , Adrian J. Thrasher MBSS, PhD , Isabelle Thuret MD , Ashutosh Lal MD, John E.J. Rasko BSc (Med), MBBS (Hons), PhD, MAICD, FFSc (RCPA), FRCPA, FRACP, FAHMS , Evangelia Yannaki MD , Shamshad Ali MS, MStat , Ge Tao PhD , Himal L. Thakar MD , Ami Deora PhD , Katiana Gruppioni MPH , Richard A. Colvin MD, PhD , Franco Locatelli MD , Janet L. Kwiatkowski MD, MSCE. “Efficacy and safety of beti-cel in pediatric patients with transfusion-dependent β-thalassemia” ASPHO Conference, April 3-6, 2024, at the Seattle Convention Center in Seattle, WA.
C. Lagresle-Peyrou^#, A. Olichon, H. Sadek, P. Roche, C. Tardy, C. Da Silva, A. Garrigue, A. Fischer, D. Moshous, Y. Collette, C. Picard, JL. Casanova, I. André, M. Cavazzana^#. An Autosomal Dominant SCID Form Due to a Gain of Function Mutation in the RAC2 Gene. 61^st Annual Meeting of the American Society of Hematology (ASH). December 2019, Orlando, USA. (^#Corresponding authors)
E. Magrin, M. Semeraro, A. Magnani, H. Puy, A. Miccio, N. Hebert, JS. Diana, F. Lefrere, F. Suarez, O. Hermine, V. Brousse, C. Poirot, P. Bourget, W. El Nemer, I. Guichard, D. Moshous, B. Neven, F. Monpoux, M. Poirée, P. Bartolucci, JF. Meritet, D. Grévent, T. Lefebvre, M. Asmal, E. Whitney, M. Gayron, W. Huang, I. Funck-Brentano, M. de Montalembert, L. Joseph, JA. Ribeil, M. Cavazzana. Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy. 61^st Annual Meeting of the American Society of Hematology (ASH). December 2019, Orlando, USA.
L. Weber, G. Frati, T. Felix, C. Wollenschlaeger, A. Casini, V. Meneghini, C. Masson, A. De Cian, A. Chalumeau, F. Mavilio, M. Amendola, I. André, A. Cereseto, W. El Nemer, JP. Concordet, C. Giovannangeli, M. Cavazzana, A. Miccio^#. Editing a γ-Globin Repressor Binding Site Restores Fetal Hemoglobin Synthesis and Corrects the Phenotype of Sickle Cell Disease Erythrocytes. 61^st Annual Meeting of the American Society of Hematology (ASH). December 2019, Orlando, USA.
JL. Kwiatkowski, AA. Thompson, JEJ. Rasko, S. Hongeng, GJ. Schiller, U. Anurathapan, M. Cavazzana, P. Joy Ho, M. Schmidt, M. Kletzel, EP. Vichinsky, B. Deary, Y. Chen, A. Petrusich, MC. Walters. Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study. 61st Annual Meeting of the American Society of Hematology (ASH). December 2019, Orlando, USA.
P. Rio, S. Navarro, R. Sanchez-Dominguez, JC. Segovia, W. Wang, E. Merino, RM. Yañez, J. Antonio Casado, YA. Gimenez, FJ. Roman-Rodriguez, O. Alberquilla, E. Galvez, R. Hladun, J. Barquinero, A. Galy, N. García de Andoín, R. López, A. Catalá, M. Cavazzana, F. Lefrere, TM. Leblanc, JD. Schwartz, R. Pujol, J. Surrallés, J. Soulier, M. Schmidt, C. Díaz de Heredia, J. Sevilla, JA. Bueren. Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy. 61st Annual Meeting of the American Society of Hematology (ASH). December 2019, Orlando, USA.
JS. Diana^#, S. Manceau, TM. Leblanc, C. Couzin, A. Magnani, E. Magrin, M. Bendavid, L. Joseph, M. Delville, S. Blanche, J. Soulier, M. Cavazzana, F. Lefrere. A New Step in Understanding of Fanconi Patients Peripheral Stem Cell Harvesting, a Bridge to Gene Therapy. 61st Annual Meeting of the American Society of Hematology (ASH). December 2019, Orlando, USA.
JL. Kwiatkowski, AA. Thompson, J. Rasko, S. Hongeng, GJ. Schiller, U. Anurathapan, M. Cavazzana, PJ. Ho, C. von Kalle, M. Kletzel, P. Leboulch, E. Vichinsky, B. Deary, M. Asmal, MC. Walters. Clinical Outcomes up to 3 Years Following Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar Hgb-204 Study. 59^th Annual Meeting of the American Society of Hematology (ASH). December 2017, Atlanta, USA.
E. Six, A. Guilloux, C. Nobles, R. Zreik, A. Lecoule, J. Everett, R. Vilette, N. Cagnard, F. Male A. Magnani, E. Magrin, L. Caccavelli C. Roudaut C. Plantier A. Galy, AJ. Thrasher, I. André-Schmutz, CC. Berry, M. Cavazzana, FD. Bushman. Diverse and Distinct Human Hematopoietic Differentiation Programs Generate by Bone Marrow and G-CSF Mobilized Hematopoietic Stem and Progenitors Cells. 59^th Annual Meeting of the American Society of Hematology (ASH). December 2017, Atlanta, USA.
A. Durandy, S. Kracker, M. Cavazzana. Primary Antibody Deficiencies: Scientific Discoveries Leading to Challenging Paradigms in a Complex Disease Landscape. 59^th Annual Meeting of the American Society of Hematology (ASH). December 2017, Atlanta, USA.
S. Kracker*, M. Semeraro, C. Cancrini*, F. Conti, S. Ehl, M. Heeg*, P. Soler-Palacin*, E. Helmer, N. Diaz*, M. Juarez*, A. Payne, G. Johnston, D. Yuill, J.M. Riethuisen, F. Höppener, M. Cavazzana*. Seletalisib, a PI3Kδ inhibitor, demonstrates biological and clinical effect in activated PI3K δ syndrome patients: results from a proof-of-concept study. 3^rd International Primary Immunodeficiencies Congress. November 2017. Dubai, UAE. (*These authors contributed equally)
E. Six, A. Lecoules, L. Caccavelli, C. Nobles, F. Male, A. Magnani, C. Roudaut, C. Plantier, E. Magrin, N. Cagnard, S. Hacein-Bey-Abina, A. Galy, AJ. Thrasher, I. André-Schmutz, A. Guilloux, CC. Berry, FD. Bushman, M. Cavazzana. A Diversity of Human Hematopoietic Differentiation Programs Identified Through In Vivo Tracking of Hematopoiesis in Wiskott-Aldrich Syndrome Patients. 20^th annual meeting of the American Society of Gene and Cell Therapy (ASGCT). May 2017. Washington, USA.
F. Amirache, O. Bernadin, A. Girard-Gagnepain, C. Costa, C. Lévy, D. Fenard, V. Asnafi, H. Sadek, C. Lagresle-Perou, I. André-Schmutz, M. Cavazzana, FL. Cosset, E. Verhoeyen. BaEV-LVs Efficiently Transduce HSCs-Derived Human Progenitor T Cells, Acceleratingt Cell Reconstitution In Vivo and Allowed Correction of X-SCID Progenitor T Cells. 20^th annual meeting of the American Society of Gene and Cell Therapy (ASGCT). May 2017. Washington, USA.
CL. Nobles, JK. Everett, CC. Berry, E. Six, F. Male, S. Reddy, K. Bittinger, S. Hacein-Bey Abina, M. Cavazzana, FD. Bushman. INSPIIRED: A Pipeline for Quantitative Detection of Newly Integrated Genomic Elements. 20^th annual meeting of the American Society of Gene and Cell Therapy (ASGCT). May 2017. Washington, USA.
CL. Nobles, CC. Berry, F. Male, SR. Gujja, E. Six, S. Hacein-Bey-Abina, M. Cavazzana, JJ. Melenhorst, JA. Fraietta, SF. Lacey, CH. June, FD. Bushman.Insertion Site Analysis as a Component of Gene Therapy Product Characterization. 20^th annual meeting of the American Society of Gene and Cell Therapy (ASGCT). May 2017. Washington, USA.
V. Poletti, S. Charrier, G. Corre, S. Martin, B. Gjata, A. Vignaud, F. Zhang, K. Buckland, I. André-Schmutz, M. Rothe, A. Schambach, SY. Pai, DA. Williams, HB. Gaspar, M. Cavazzana, AJ. Thrasher, F. Mavilio. Preclinical Development of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1). 20^th annual meeting of the American Society of Gene and Cell Therapy (ASGCT). May 2017. Washington, USA.
C. Antoniani, V. Meneghini, A Lattanzi, T. Felix, O. Romano, E. Magrin, L. Weber, G. Pavani, TJ. Cradick, AS. Lundberg, M. Amendola, W. El Nemer, M. Cavazzana, F. Mavilio, A. Miccio. Induction of Fetal Hemoglobin Synthesis in Human Erythroblasts by CRISPR/Cas9-Mediated Editing of the β-Globin Locus. 20^th annual meeting of the American Society of Gene and Cell Therapy (ASGCT). May 2017. Washington, USA.
E. Six, V. Gandemer, A. Magnani, C. Nobles, J. Everett, F. Male, C. Plantier, I. Hmitou, M. Semeraro, E. Magrin, L. Caccavelli, A. Trinquand, V. Asnafi, JM. Lapierre, C. Tuchmann-Durand, I. André-Schmutz, S. Hacein-Bey-Abina, S. Romana, E. Macintyre, FD. Bushman, A. Fischer*, M. Cavazzana*. LMO2 Associated Clonal T Cell Proliferation 15 Years After Gamma-Retrovirus Mediated Gene Therapy for SCIDX1. 20^th annual meeting of the American Society of Gene and Cell Therapy (ASGCT). May 2017. Washington, USA. (*These authors contributed equally)
JS. Diana, D. Moshous, M. Castelle, ML. Fremond, A. Magnani, E. Elkaim, C. Briand, M. Bendavid, E. Magrin, A. Fischer, M. Cavazzana, S. Blanche, B. Neven, Benedicte. Haploidentical transplantation with post-transplant cyclophosphamide for pediatric non-malignant disorders. European Society for Blood and Marrow transplantation (EBMT). March 2017. Marseille, France.
A. Galy, G. Santilli, S. Stein, J. Schwaeble, U. Siler, A. Magnani, S. Pouillot, K. Kuehlcke, M. Schmidt, T. Paprotka, G. Honnet, F. Mavilio, M. Grez, H. Serve, J. Reichenbach, S. Blanche, H.B. Gaspar, M. Cavazzana, A.J. Thrasher. Net4CGD: advancing gene therapy for a rare primary immune deficiency. 3^rd International Rare Disease Research Consortium (IRDiRC). February 2017. Paris, France.
below are another 255 abstract..

CONGRESS ORGANIZATION AND SESSION CHAIRS

International

HIV/AIDS Europe Roundtable, March 20, 2018, Brussels, Belgium
1^st International Symposium on Inner Ear Therapies, Round table Co-chair “From bench to bedside: Insights for a good clinical trial design”. November 2017. Marrakesh, Morocco.
5^th World Cord Blood Congress, Co-organizer. March 2015. Monaco.
39^th European Society for Blood and Marrow Transplantation (EBMT) Annual Meeting, Co-chaiperson of session «Gene therapy for immunodeficiencies and metabolic disease”. April 2013. London, UK.
15^th Biennal Meeting of the European Society for Immunodeficiencies (ESID), Co-organizer of the “Gene Therapy” working party session. October 2012. Florence, Italy.
38^th European Society for Blood and Marrow Transplantation (EBMT) Annual Meeting, Co-chairperson of plenary session 2 “Cellular Therapy”. April 2012. Geneva, Switzerland.
3^rd World Cord Blood Congress, Co-chaiperson of the “Panel Discussion on Adoptive Immunotherapy”. October 2011. Rome, Italy.
London – Padua – Paris Labs Network, 1^st International Workshop on Regenerative Medicine in Pediatrics, Co-organizer and Moderator. February 2011. Padua, Italy.
52^nd American Society of Hematology Annual Meeting (ASH), Co-chair of oral session on “Lymphocytes, lymphocyte activation and immunodeficiency, including HIV and other infections: immunodeficiency, autoimmunity and immunomodulation”. December 2010. Orlando, Florida, USA.
European Society for Blood and Marrow Transplantation (EBMT) Inborn Errors Working Party Meeting, Co-organizer, San Servolo. November 2010. Venice, Italy.
5^th Stem Cell Clonality & Genotoxicity Retreat, Co-chairwoman of session on “Globin gene therapy. November 2009. Hannover, Germany.
International Symposium on Immunotherapy and Immunodeficiency, Co-chairwoman of session on “Imunodeficiencies and Gene Therapy”. November 2009. Heidelberg, Germany.
35^th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), Co-chair of oral session on “Cellular and Gene Therapies”. March-April 2009. Göteborg, Sweden.
CSLS Behring Immunology Workshop, Moderator, Session on “Principles and Progresses in Immunodeficiencies”. January 2009, Monaco.
Annual Meeting of European Society for Blood and Marrow Transplantation (EBMT), Chairwoman of the working party group “Hereditary Diseases” for the European Group for Blood and Marrow Transplantation”. March 2005. Prague, Tcheque Republic.
European Society for Blood and Marrow Transplantation (EBMT), Chair of the Immunodeficiencies session. June 2004. Noordwijkerhout, The Netherlands.

National

what impact on the progression and management of diseases” February 8, 2023. Imagine Institute, Paris, France.
January 23, 2023. Imagine Institute, Paris, France.
November 2022, Imagine Institute, Paris, France
March 16, 2022. Imagine Institute, Paris, France.
February 28, 2022. French Presidency of the European Union, France
February 8, 2022. Imagine Institute, Paris, France.
March 28, 2021, Imagine Institute, Paris, France

General public conference on gene therapy “When DNA becomes medicine”, September 2019, Imagine Institute, Paris, France.
International DIM Gene Therapy Symposium, “Gene Therapy for Rare Monogenic Diseases”. February 2019. Imagine Institute, Paris, France.
Gene Therapy Partnering Day, in collaboration with Imagine Institute and Medicen. 17 September 2018, Paris, France
Pfizer Dinner Debate, “Gene Therapy, a Medical Revolution, but What Expectations for Patients?” March 6, 2018, Paris, France.
Prevention and Screening of Sickle Cell Disease. April 2017. Imagine Institute, Paris, France. Necker Enfants-Malades Pierre Royer Seminar of Pediatric Immunology, Haematology and Rheumatology, “Gene therapy for hemoglobinopathies”. April 2016. Paris, France.
Necker Enfants-Malades Pierre Royer Seminar of Pediatric Immunology, Haematology and Rheumatology, “Gene therapy for hemoglobinopathies: preliminary results and expected progress”. April 2014. Paris, France.
2^nd Meeting Network ANRS Gene Therapy for HIV patients with lymphonia. January 2014. Imagine Institute, Paris, France.
Imagine Institute – Pierre Royer Seminar of Pediatric Immunology, Haematology and Rheumatology, Co-organizer and Chairwoman of the Invited Conferences session. March 2013. Paris, France.
1^st Rencontres Maladies Rares, Chaiperson of the session « Thérapeutiques Innovantes ». June 2012. Paris, France.
Workshop Monacord-Eurocord « Observatoire de la Drépanocytose ». Co-chairperson of session « Registre des femmes enceintes ». April 2012, Monaco.
Necker Enfants-Malades Pierre Royer Seminar of Pediatric Immunology, Haematology and Rheumatology, Co-organizer. March 2012. Paris, France.
37^th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT), Member of the National Scientific and Organization CommitteeCo-chairperson of session “Immune reconstitution after HSCT”. April 2011. Paris, France.
37^th Meeting of the European Society for Blood and Marrow Transplantation (EBMT), Miltenyi Corporate Satellite Symposium “Cellular Therapy: State of the art and future perspectives”, Chairperson. April 2011. Paris, France
Necker Enfants-Malades Pierre Royer Seminar of Pediatric Immunology, Haematology and Rheumatology, Co-organizer and Chairwoman of session on “Medullar aplasia”. March 2011. Paris.
Necker Enfants-Malades Pierre Royer Seminar of Pediatric Immunology, Haematology and Rheumatology, Co-organizer and Chairwoman. March 2010. Paris, France.
Necker Enfants-Malades Pierre Royer Seminar of Pediatric Immunology, Haematology and Rheumatology, Co-organizer. March 2009. Paris, France.
Annual Seminar of Pediatric Immunology, Hematology & Rhumatology, Hospital Necker Enfants-Malades, Co-organizer, Moderator of session on “News in Hematology”, April 2008, Paris
1^st Joint Meeting of European National Societies of Immunology, 16^th European Congress of Immunoloy, Co-Chairwoman of Session of “Prions & Gene Therapy”. September 2006. Palais des Congrès, Paris, France
Pasteur Institute Euroconferences on “Gene and Cell Therapy”-, Co-organizers: Candotti F, Cavazzana-Calvo M, Heard JM, Roncarolo MG; Moderator of session on stem cells and therapy. December 2005. Paris, France
Collège de France, Chair of Experimental Medicine Seminar: Role of gene therapy in the treatment of hereditary diseases of the lymph system. March 2003. Paris, France.

INTERVIEWS

Interview for Insights on Pre-Transplant Drug Development – November 13, 2024
Interview-collaboration on Rare Diseases and Orphan Drugs Project – 8 octobre 2024 – with Clarivate, a Philadelphia based leading Healthcare
Consultation – Sickle Cell Disease Discussion (NYBC Ventures) – October 4, 2024
Interview with MIT-Harvard student group – Topic: Interview on Gene Therapy wih Prof Cavazzana (Jul 9, 2024)
Interview DG SANTE/HaDEA – Hospital Exemption Study (MTI PP): Study on Hospital Exemption (MTI-PP) in relation to the implementation of the Advanced Therapy Medicinal Products (ATMP) regulation – May 2024
Telephone interview with Les Echos Yann Verdo on RNA: messenger RNA with an educational angle on advances in medicine – Thursday, January 25, 2024
ASH Clinical News on the cost effectiveness of gene therapy for sickle cell disease and access to advanced therapies. May 25, 2023
Curing Genetic Diseases. Practitioner’s Review No. 999, April 2018
A child grafted with his own genetically modified skin: “An extraordinary step”, November 9, 2017

https://www.lci.fr/sante/un-enfant-greffe-avec-sa-propre-peau-genetiquement-modifiee-quels-espoirs-pour les-malades-etape-extraordinaire-2069926.html

Sickle cell disease: a 13-year-old child successfully treated with gene therapy, October 23, 2017

https://www.sciencesetavenir.fr/sante/drepanocytose-un-enfant-de-13-ans-traite-avec-succes-par-la-therapie-genique_117586

The day when… Gene therapy saved a 13-year-old boy. Le Parisien, August 12, 2017
Correction of defective genes in humans: “Morality also means taking into account parents affected by genetic mutations.” France Info, August 3, 2017
Imagine, doctors from all over the world in the heart of Paris. La Croix, May 31, 2017
Effective gene therapy against sickle cell disease. Le Figaro.fr, March 2, 2017
Sickle cell disease: gene therapy successful in the first patient treated. Le Parisien, March 1, 2017
Sickle cell disease: first cure through gene therapy. Libération, March 1, 2017
Gene therapy ‘cures’ boy of blood disease that affects millions. New Scientist, 1 March 2017
50 years of Inserm – Professor Marina Cavazzana: she took babies out of their bubble. Le Point, March 27, 2014
Marina Cavazzana-Calvo, the passionaria of gene therapy. La Dépêche, April 7, 2013
Marina Cavazzana, pioneer of gene therapy, October 2017

https://www.sciencesetavenir.fr/sante/video-marina-cavazzana-pionniere-de-la-therapie-genique_117239