Dr. Donald Kohn headshotA team of researchers from the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA and Orchard Therapeutics Limited have been awarded $20 million by the California Institute for Regenerative Medicine’s governing board. An additional $18.1 million of co-funding will be provided by Orchard Therapeutics Limited.

Over five years, 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, which is fatal if left untreated.

CIRM representative Dr. Gil Sambrano noted during the board’s meeting today that this clinical trial “could be the first cell gene therapy approved by FDA.”

Led by clinician-scientist Dr. Donald Kohn, the ADA-SCID trials have cured 30 out of 30 babies to date through a stem cell gene therapy method that is currently only available at UCLA and a second site in London, U.K. Babies with the condition and their families have traveled to UCLA for this life-saving treatment from places as far away as Canada and Lebanon. In May 2016, Orchard Therapeutics Limited was formed to expand physician access to the technology, which would ideally allow patients to receive the treatment closer to home.  

“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. “As this treatment for ADA-SCID continues to move forward, I hope the momentum will result in better treatments for other blood diseases we’re working on, such as sickle cell disease.”

The treatment, developed by Dr. Kohn over 30 years of research, removes the baby’s own blood-forming stem cells and fixes the broken genes to eliminate the disease-causing mutations and the associated debilitating symptoms. The corrected cells are then transplanted back into the baby in what is called an ‘autologous’ transplant.

With the new funding, Dr. Kohn and Orchard Therapeutics Limited will test a method that freezes the blood stem cells after they have been genetically corrected.

Freezing the cells will allow for:

  • Better testing of the cells prior to transplant;
  • More time to customize treatment plans based on patient needs; and
  • The transportation of cells from the gene correction facility back to a transplant center closer to a patient’s home.

Kohn is a paid consultant on the Orchard Therapeutics Limited Scientific Advisory Board, and The Regents of the University of California have licensed intellectual property to Orchard Therapeutics Limited.

In this video, Dr. Kohn discusses a method that uses a patient's own blood-forming stem cells and a gene editing technique to correct genetic mutations that cause blood diseases, such as sickle cell and “bubble baby” disease.

Media Contact: 

Mirabai Vogt-James
(310) 983-1163
mvogt@mednet.ucla.edu