CIRM grants Orchard, UCLA $20M for ADA-SCID trial

Gene therapy company Orchard Therapeutics Ltd. (London, U.K.) said the California Institute for Regenerative Medicine (CIRM) awarded the company and University of California Los Angeles researchers $20 million to conduct a clinical trial of an autologous ex vivo lentiviral gene therapy to treat severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency.

Orchard said more than 40 patients have already received the therapy at UCLA and the Great Ormond Street Hospital in London. All have survived and the treatment has been shown to restore immune function with a favorable safety profile, the company said...