Friday, August 23, 2019

The Search for a Cure for the Bubble Boy Disease: Orchard Therapeutics and the California Stem Cell Agency

Alliance for Regenerative Medicine video

Orchard Therapeutics, a British firm in which the California stem cell agency is directly supporting with $8.5 million, reports that it is "well underway" with its plans for a major facility in the San Francisco Bay area. 

Orchard says that it is on schedule to open the 150,000-square-foot plant in Fremont, Ca., in 2021, according to an article on BioProcess International.  

The move is part of a broad effort by Orchard to commercialize genetic therapies based on research pursued for decades by Donald Kohn of UCLA. California's stem cell research program has pumped $52 million into Kohn's research, which has saved the lives of more than 50 babies in (ADA-SCID) clinical trials.

Kohn has targeted what is often known as the bubble boy syndrome, a rare, fatal immune deficiency disorder (adenosine deaminase-deficient severe combined immunodeficiency -- ADA-SCID).  

UCLA has licensed Kohn's work to Orchard and will receive compensation at some point. CIRM is also slated to receive compensation should a product be developed. Kohn is one of the scientific founders or the Orchard and is a scientific adviser to the firm. 

The BioProcess article last week by Dan Stanton quoted Ran Zheng, Orchard's chief technical officer, as saying, 
“The diseases we target affect patients around the world, which require us to have the infrastructure to deliver gene therapies globally. We are therefore building a commercial-scale manufacturing infrastructure and leveraging technologies that will allow us to deliver our gene therapies globally and in a fully-integrated manner.”

The Fremont facility represents only part of Orchard's presence in California. Ron Leuty of the San Francisco Business Times reported that the firm leases 14,138 square feet in Menlo Park for labs and offices and 4,472 square feet for development labs and offices in Foster City. 

Orchard's stock price closed at $13.67 yesterday. Its 52-week high was $21.64 and the 52-week low was $8.65.

(Editor's note: This item has been altered from an earlier version to be more specific about the number of babies whose lives have been saved in clinical trials involving Kohn's work. The number now exceeds 50.) 

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