Marina Cavazzana

Marina Cavazzana is a professor of Paediatric Immunology at the Necker-Enfants Malades Hospital and the Imagine Institute, as well as an academic at Paris Descartes University. She was awarded the Irène Joliot-Curie Prize in 2012 and elected to the National Academy of Medicine in 2019.

Early life and education Cavazzana was born in Venice. Her father worked on the railway and her mother was a teacher. She studied medicine in Padua, where she completed her residency at the University of Padua. She moved to Paris, where she earned her doctorate at the Paris Diderot University studying bone marrow transplants with Élaine Gluckman at the Hôpital Saint-Louis. She established the first Clinical Investigation Center in the Assistance Publique – Hôpitaux de Paris, which is the only French academic department is authorised to produce cell and gene therapies.

Her research considers the haematopoietic immune system and gene therapy. She has designed clinical trials based on lentiviral vector in gene therapy and pioneered the use of haematopoietic stem cell gene therapy for genetic immune and haematopoietic disorders. Cavazzana was the first doctor to successfully treat a boy with sickle cell disease, a severe form of hereditary chronic anemia. Sickle cell disease is a blood disorder that results from an abnormality in red blood cells. It occurs due to a mutation on the HBB gene, and results in a sickle-like deformity of red blood cells. It is the most common genetic disorder in France. The first gene therapy trail of young boys with X-linked severe combined immunodeficiency was performed by Cavazzana in collaboration with bluebird bio in 2000. When it was established that two of the boys developed leukaemia from the gene therapy, Cavazzana coordinated the medical efforts of biologists, virologists, clinicians and regulators to develop novel medical therapies. Children who are born with Thalassemia Major often develop anaemia within their first years of life. Wiskott–Aldrich syndrome is an immunodeficiency associated with microthrombocytopenia. The child with sickle cell disease had a restored clinical and biological phenotype within sixth months of the gene therapy. The law requires close family members to be informed when individuals are at risk of developing a genetically transmitted illness.

Awards and honours 2006 : Prix de Recherche, clinique et thérapeutique 2012 : Prix Irène-Joliot-Curie de la Femme scientifique de l'année 2016 : Prize of l'Académie nationale de médecine 2017 : Fondation pour la recherche médicale Fondation Guillaume-Piel Award 2017 : American Society of Hematology Ernest Beutler Lecture and Prize for Clinical Science 2019 : University of Zurich Honorary Doctorate 2019 : Elected to the National Academy of Medicine