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Orchard abandons promising gene therapy for rare immune disorder

Dive Brief:

  • Orchard Therapeutics has officially abandoned development of a promising treatment for a rare genetic disorder, terminating its license from the University of California Los Angeles and the University College London.


  • The company gave no reason for its decision in a short regulatory filing Friday. In a May 28 letter to partners and advocates, Orchard CEO Bobby Gaspar said the London-based biotech had encountered technical issues in manufacturing commercial-grade product and couldn’t find a “viable path forward” after considering possible solutions and seeking new partners.


  • Gaspar also referenced the “strong preference” in the patient advocate community that the treatment, dubbed OTL-101, be returned to academic partners. Orchard will help facilitate compassionate use of the therapy and make data and other resources available, he said.


Dive Insight:

Orchard’s decision is suggestive of the difficulty drugmakers can face in turning complex, but promising rare disease treatments into a sustainable business. The termination wasn’t unexpected; Orchard said last May it was reducing staff and reordering its priorities, reducing investment in OTL-101 as a result.

The therapy is designed to treat ADA-SCID, a genetic condition in which children are born without a properly functioning immune system. The group of diseases known as severe combined immunodeficiency, or SCID, first drew wide attention decades ago because of David Vetter, a boy born with X-linked SCID who lived in a bubble for 12 years as doctors tried to figure out how to cure him.

ADA-SCID is caused by a gene mutation that affects a critical protein called adenosine deaminase, which is needed to keep toxins in the body from destroying disease-fighting white blood cells. Stem cell transplants from matched, related donors can be effective, but most patients don’t have such donors available to them.

OTL-101, by comparison, is designed to correct the problem by inserting working copies of the gene into a patient’s blood stem cells.

Just weeks ago, Orchard touted research in The New England Journal of Medicine supporting the use of the therapy. The study included 30 patients treated with OTL-101 and found that all of them were still alive after two years.

Orchard’s decision last year to put OTL-101 on the back burner left patients with no way to access a seemingly effective therapy for a condition that can kill children before they reach the age of one. So, the company’s latest announcement is “wonderful news,” according to the California Institute for Regenerative Medicine, or CIRM, which helped fund development of the treatment.

“This means that UCLA will now be able to begin the process of applying to the Food and Drug Administration for permission to start an expanded use protocol to treat patients,” CIRM CEO Maria Millan said in a May 28 statement.

UCLA scientist Donald Kohn, who developed the therapy, told the Los Angeles Times that he will start the FDA process for compassionate use as soon as possible. UCLA and UCL will also seek a new commercial partner, Kohn said.