The Gene Therapy 'Quagmire:' Multimillion- Dollar Costs and Untreated Patients with Rare Diseases
By David Jensen,
The California Stem Cell Report
| 08. 01. 2023
Jason Mast is a former English teacher who now focuses on such things as multimillion-dollar gene therapies and whether they ever reach patients.
Mast writes for STAT, the respected online biomedical news service. On Monday, he encapsulated the “gene therapy crisis” in 115 words:
“Jennifer Puck has successfully treated 10 children with a gene therapy for a fatal disorder that decimates their immune system. But she has no idea how to get her drug approved and frankly is running out of ideas.
“‘I wish I had a clue about where to go from here,’ said Puck, an immunologist at University of California, San Francisco….
“The problem is simple: Size,” Mast wrote. “Puck’s therapy is for a disease, Artemis-SCID, that affects just two to three new U.S. patients every year — far too few for a company to generate a profit, or to even run the kind of studies regulators usually demand before approving drugs.”
All in all, Mast wrote: “a quagmire born of the field’s own success.”
The 115 words were just a start in a lengthy piece...
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