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
By Diaa Hadid and Shweta Desai, NPR | 01.29.2026
MUMBRA, India — The afternoon sun shines on the woman in a commuter-town café, highlighting her almond-shaped eyes and pale skin, a look often sought after by couples who need an egg to have a baby.
"I have good eggs,"...
By George Janes, BioNews | 01.12.2026
A heart attack patient has become the first person to be treated in a clinical trial of an experimental gene therapy, which aims to strengthen blood vessels after coronary bypass surgery.
Coronary artery bypass surgery is performed to treat...
By Staff, ScienceDaily | 01.05.2026
Scientists at UNSW Sydney have developed a new form of CRISPR technology that could make gene therapy safer while also resolving a decades-long debate about how genes are switched off. The research shows that small chemical markers attached to DNA
...
Following a long-standing CGS tradition, we present a selection of our favorite Biopolitical Times posts of the past year.
In 2025, we published up to four posts every month, written by 12 authors (staff, consultants and allies), some in collaboration and one simply credited to CGS.
These titles are presented in chronological order, except for three In Memoriam notices, which follow. Many more posts that are worth your time can be found in the archive. Scroll down and “VIEW...
