A new trial in gene therapy led by UCLA in partnership with Genethon

10 children with an immunodeficiency disorder, commonly referred to as X-linked CGD, will be enrolled to receive a stem cell gene therapy, due to Dr Kohn’s team and Genethon.

Genethon, AFM-Téléthon’s laboratory research, engineered the virus delivery system designed to transport the gene therapy into patients’ blood-forming stem cells. Genethon is sponsoring a parallel European clinical trial that is currently open and recruiting in three cities (London, Frankfurt and Zurich) and is also preparing to open in Paris. The european trial sites utilize  the same gene therapy to be used in Dr.Kohn’s trial.

chronic granulomatous disease, which prevents white blood cells from effectively killing foreign invadeers such as bacteria, fungi and other microorg anisms, is an inherited blood disorder affecting approximately 1 in 200 000 persons in the U.S.

This trial receives $7.4 million inClinical trial led by UCLA receives $7.4 million.

More information with the press release.