Marina Cavazzana

Professor Marina Cavazzana, MD, PhD, is a globally recognised clinician-scientist and pioneer in gene therapy and hematopoietic stem cell research. She trained in medicine and specialized in hematology before undertaking advanced research in gene therapy, ultimately establishing herself as one of the leading figures in translating genomic medicine into clinical practice.
Professor Cavazzana serves as Director of Inserm U1163 at Institut Imagine, where she leads research on genetic diseases, stem cell biology, and novel therapeutic platforms. She holds a professorship in Hematology at the Université de Paris and practices as a Senior Physician at Necker–Enfants Malades Hospital (Assistance Publique – Hôpitaux de Paris), where her clinical expertise focuses on severe combined immunodeficiencies and other life-threatening monogenic disorders.
Her laboratory’s seminal contributions include the development and clinical implementation of successful gene therapy protocols for SCID (including ADA-SCID and X-linked SCID) and other hematologic genetic diseases. Through collaborative networks spanning basic science, translational development, and clinical trials, Professor Cavazzana has helped demonstrate the curative potential of gene-corrected hematopoietic stem cells—advancing sustained clinical benefit in patients with previously untreatable conditions.
She has published extensively in leading journals including Nature, Science, and Blood, and her work has shaped international clinical standards for gene therapy and stem cell transplantation. Beyond research, Professor Cavazzana has played leadership roles in scientific societies, advisory councils, and regulatory committees guiding advanced therapy development and ethical governance.
Her achievements have garnered major international recognition and reflect a career committed to integrating cutting-edge genomic science with compassionate clinical care, thereby transforming the therapeutic landscape for patients with rare genetic diseases.