An update on the stem cell gene therapy clinical trial for ADA-SCID

Orchard Therapeutics has transferred back to UCLA the FDA license to conduct the clinical trial of an experimental blood stem cell gene therapy for children with a rare and deadly inherited disorder that leaves them without an immune system.

Developed by a team of researchers from UCLA and University College London, the therapy has successfully treated 48 of 50 children born with severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID. The FDA license is formally known as an Investigational New Drug (IND) application.

UCLA worked closely with the California Institute for Regenerative Medicine to return the state stem cell agency’s grant funding from Orchard Therapeutics to UCLA to fund the trial. The remaining grant funding will support treatment for three or four patients. UCLA will submit a follow-on grant to CIRM to secure the resources necessary to treat the rest of the patients who have been waiting to undergo this therapy.

The clinical trial will continue to be led by Dr. Donald Kohn, a distinguished professor of microbiology, immunology and molecular genetics and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. 

“We hope to be able to initiate the clinical trial and enroll and treat the first waiting patient within the next four months,” said Kohn, who is also a professor of pediatrics and molecular and medical pharmacology at the David Geffen School of Medicine at UCLA. “We are grateful to all parties for their collaborative efforts to make this life-saving therapy accessible to patients.”

While the expanded access clinical trial is ongoing, UCLA’s Technology Development Group will work to obtain any residual rights from Orchard and to identify an industry partner that has the qualifications, interest and successful track record to make the treatment widely available to patients.