Fulvio Mavilio

Genethon is a biotherapy research and development firm, which engages in the design, development, and production of gene therapy treatments for rare diseases. It specializes in neuromuscular diseases, immune system or blood disorders, eye disorders, and liver and nervous system diseases. The company was founded in 1990 and is headquartered in Evry, France.

Astellas Gene Therapies, Inc. is a biotechnology company, which engages in the development and commercialization of gene therapy products for patients with serious and rare diseases caused by single gene defects. The company was founded by Matthew R. Patterson and Thomas J. Schuetz on November 13, 2012 and is headquartered in San Francisco, CA.

AGC Biologics SpA engages in the research, development, manufacturing, and clinical validation of cell and gene therapies for the treatment of cancer and rare diseases. Its approach is based on finding drugs that target the reduction of tumor mass in the acute stage, as well as on the provision of selective therapies to eliminate the residual disease. It also conducts cell and gene therapy projects in collaboration with third parties, offering resources and expertise covering preclinical to Phase III trial activities. The company was founded by Claudio Bordignon on July 11, 1996 and is headquartered in Milan, Italy.

The American Society of Gene & Cell Therapy is a medical and professional organization that represents researchers and scientists devoted to the discovery of gene and cell therapies. The American company was founded by Mark A. Kay.

Orchard Therapeutics Plc is a biopharmaceutical company, which engages in discovering, acquiring, developing and commercializing gene therapies for patients with rare disorders. The company focuses on its autologous ex vivo gene therapy approach on three therapeutic rare disease franchise areas: primary immune deficiencies, neurometabolic disorders, and hemoglobinopathies. Its portfolio includes Strimvelis, a commercial-stage gamma retroviral-based product for the treatment of Adenosine deaminase deficiency (ADA-SCID). The company was founded by Nicolas Koebel and Andrea Spezzi in August 2018 and is headquartered in London, the United Kingdom.

Smart Immune SAS is a clinical-stage biotechnology company based in Paris, France. Smart Immune is developing Protcell, a thymus-empowered T cell progenitor therapy platform to re-arm the immune system against cancer and infection, enabling next-generation allogeneic T cell therapies for all. The French company aims to improve outcomes for patients with life-threatening diseases such as high-risk blood cancers and primary immunodeficiencies. Smart Immune has ongoing collaborations with leading institutions in the EU and the US. Smart101 is already in phase I/II clinical trials in the EU and the US, looking at the acceleration of complete immune recovery in patients fighting cancer and infection and undergoing allogeneic hematopoietic stem cell transplantation. Smart Immune is also developing therapies using gene-modified T cell progenitors through its Protcell platform to provide targeted treatments against cancer, like persistent off-the-shelf CAR T cells. The company is headquartered at Biotech Santé. The company was founded in 2017 by Karine Rossignol, Marina Cavazzana, Isabelle André, with Karine Rossignol as the CEO since 2017.