The California Institute for Regenerative Medicine provided a $20 million grant to two institutions working on an experimental gene therapy.
Orchard Therapeutics and scientists from UCLA’s Broad Stem Cell Research Center will use this funding to run a clinical trial testing their treatment for Severe Combined Immunodeficiency caused by adenosine deaminase deficiency also known as ADA-SCID.
This condition produces mutations that lead to a serious shortage of white blood cells. Infants tend to get diagnosed with this rare condition leaving them vulnerable to life-threatening infections.
Orchard’s drug works by taking the patient’s own stem cells, modifying them with a functioning copy of the missing or damaged gene, and then inserting this new cocktail back into the patients body. The idea is that these altered cells would eliminate the need for a donor, according to the company’s statement.
A total of 40 patients have been tested with this therapy so far, reported FierceBiotech. All participants are still alive with immune function safely restored.
GlaxoSmithKline is working on a similar treatment for this disease, which was granted European approval earlier this year.
Filed Under: Drug Discovery
