Orchard Therapeutics, a british biotechnology company, announced a strategic alliance with Généthon, French R&D organization, to develop gene therapy for X-linked chronic granulomatous disease (“X-CGD”). Généthon is a non-profit research and development organization whose mission is to develop gene therapies for orphan genetic diseases from research to clinical validation.
X-linked CGD is a primary immune deficiency resulting from a mutation on the X-chromosome and affecting mainly boys. Patients suffering from this disease are susceptible to severe life-threatening bacterial and fungal infections and excessive inflammation characterized by granuloma formation in any organ, for instance, the gastrointestinal and genitourinary tract. Repeated episodes of infection and inflammation severely reduce the life expectancy and quality of life of patients.
Under the terms of the agreement, Orchard has obtained an exclusive option to license Généthon’s rights and know-how related to the G1XCGD lentiviral vector, including rights to the data generated under ongoing clinical trials supported by Généthon in the US and in Europe to assess the safety and efficacy of CD34+ autologous stem cells transduced with G1XCGD for the treatment of X-CGD. The program has already received an Orphan Drug Designation in Europe where the trial has been supported by the FP7 Health program of the European Commission through the Net4CGD collaborative project. As part of the agreement, Yposkesi, the industrial platform for gene and cell therapy created by Généthon, AFM-Telethon and the Sociétés de Projets Industriels (“SPI”) from BPI-France (Banque Publique d’Investissement) will manufacture a number of G1XCGD lentiviral batches for Orchard.
Mark Rothera, president and CEO of Orchard said: “This alliance with Généthonbroadens our footprint in primary immune deficiencies. It adds an important second clinical stage program to our lead program OTL-101 in ADA-SCID (adenosine deaminase severe combined immunodeficiency) which is currently at the pre-registration stage. We look forward to working with Généthon and YposKesi and are excited about the much-needed patient benefits this alliance will deliver.”
Frédéric Revah, CEO of Généthon, commented :“As a R&D organisation created by a patient association, our objective is to ensure that the innovative drugs we develop are made available to patients. We are pleased to collaborate with Orchard to continue the development of this program, which could lead, pending on results, to market authorization, and thus provide patients affected with this life-threatening disease access to the therapy”