Rare Daily Staff

Orchard Therapeutics said it entered into a strategic alliance with Généthon, the non-profit research and development organization developing gene therapies for orphan genetic diseases, to develop gene therapy for the life-threatening immune deficiency X-linked chronic granulomatous disease.

X-linked CGD results from a mutation on the X-chromosome and affects 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. 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 therapy under development including rights to the data generated under ongoing clinical trials supported by Généthon in the United States and in Europe. The program has already received an Orphan Drug Designation in Europe

This alliance with Généthon broadens our footprint in primary immune deficiencies,” said Mark Rothera, president and CEO of Orchard. “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.”

December 18, 2017