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...
Related Articles
GeneWatch UK has prepared a briefing on the genetic modification of nature for the International Union for Conservation of Nature (IUCN) Congress in October 2025
The upcoming Congress claims to be “where the world comes together to set priorities and drive conservation and sustainable development action.” A major concern for those on the outside is that the Congress may advance plans to develop and encourage the use of synthetic biology in nature conservation. This could at first glance sound like...
By Aaron Ginn, The Washington Post | 09.12.2025
Earlier this year, I had dinner in D.C. with Jensen Huang, the president and chief executive of Nvidia. At one point, he said something that struck me: “Why is everyone here so negative?”
He wasn’t referring to the economy...
By Roni Caryn Rabin, The New York Times | 08.25.2025
Scientists have dreamed for centuries about using animal organs to treat ailing humans. In recent years, those efforts have begun to bear fruit: Researchers have begun transplanting the hearts and kidneys of genetically modified pigs into patients, with varying degrees...
The Center for Genetics and Society is delighted to recommend the current edition of GMWatch Review – Number 589. UK-based GMWatch, a long-standing ally, was founded in 1998 by Jonathan Matthews as an independent organization seeking to counter the enormous corporate political power and propaganda of the GMO industry and its supporters. Matthews and Claire Robinson are its directors and managing editors.
CGS works to ensure that social justice, equity, human rights, and democratic governance are front...
