Updated on 23 November 2016
Orchard Therapeutics Limited, a clinical-stage biotechnology company, dedicated to bringing transformative gene therapies to patients with serious and life-threatening orphan diseases and researchers from the UCLA Broad Stem Cell Research, led by Orchard Scientific Advisory Board member Dr. Donald Kohn have been awarded $20 million by the California Institute for Regenerative Medicine's governing board (CIRM).
The grant will fund a new clinical trial for Severe Combined Immunodeficiency caused by adenosine deaminase deficiency, commonly known as ADA-SCID or "bubble baby" disease. ADA-SCID is a rare inherited disorder of the immune system. ADA-SCID is caused by mutations in the gene encoding for the enzyme adenosine deaminase, which result in a severe deficiency in white blood cells and life-threatening infections. In the absence of treatment, ADA-SCID is fatal within the first months of life.
To date, over 40 ADA-SCID patients have been treated in clinical trials with autologous ex-vivo lentiviral gene therapy at UCLA, Los Angeles and at the Great Ormond Street Hospital (GOSH) in London, UK. All patients have survived (100% overall survival) and the treatment has been shown to restore patients' immune function, with a favourable safety profile.
The treatment uses the patient's own stem cells which are modified with a functioning copy of the missing or faulty gene before being transplanted back into the patient's body. The use of the patient's own cells (autologous) removes the need to search for a matching stem cell donor, which can take months or even years.
"We are very grateful for this funding and for all the patients who have already gone through our clinical trials," said Dr. Kohn, a professor of pediatrics in the UCLA David Geffen School of Medicine, professor of microbiology, immunology and molecular genetics in Life Sciences at UCLA, member of the UCLA Children's Discovery and Innovation Institute at Mattel Children's Hospital and the principal investigator on the trial.
Dr. Alexander Pasteur, Orchard's interim Chief Executive Officer, added "funding from CIRM will allow UCLA and Orchard Therapeutics to accelerate the development program with ex-vivo autologous lentiviral gene therapy, which could potentially greatly benefit patients."