Orchard Therapeutics and researchers from the University of California Los Angeles Broad Stem Cell Research have been awarded $20 million from the California Institute for Regenerative Medicine’s governing to fund a clinical trial for adenosine deaminase deficiency.
The rare, inherited disorder is caused by mutations in a gene that encodes for adenosine deaminase. This results in a white blood cell deficiency and life-threatening infections. Without treatment, ADA-SCID is fatal within the 1st months of a person’s life.
More than 40 ADA-SCID patients have been treated around the world using autologous ex-vivo lentiviral gene therapy. All of the patients have survived and the therapy has safely restored patients’ immune function, according to the 2 groups.
The therapy uses a patient’s stem cells, removing the need for a matching stem cell donor. The stem cells are modified with a copy of the missing or faulty gene before being transplanted back into the patient.
“We are very grateful for this funding and for all the patients who have already gone through our clinical trials,” principal investigator Dr. Kohn said in prepared remarks.
“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,” Orchard’s interim CEO Dr. Alexander Pasteur added.