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 Anumita Kaur [cites CGS’ Katie Hasson], The Washington Post | 03.25.2025
Genetic information company 23andMe has said that it is headed to bankruptcy court, raising questions for what happens to the DNA shared by millions of people with the company via saliva test kits.
Sunday’s announcement clears the way for a new...
By Peter Wehling, Tino Plümecke, and Isabelle Bartram
| 03.26.2025
This article was originally published as “Soziogenomik und polygene Scores” in issue 272 (February 2025) of the German-language journal Gen-ethischer Informationsdienst (GID); translated by the authors.
In mid-November 2024, the British organization Hope not Hate published its investigative research ‘Inside the Eugenics Revival’. In addition to documentating an active international “race research” network, the investigation also brought to light the existence of a US start-up that offers eugenic embryo selection. Heliospect Genomics aims to enable wealthy couples to...
By Frank Landymore, Futurism | 03.18.2025
You can only throw so much money at a problem.
This, more or less, is the line being taken by AI researchers in a recent survey. Asked whether "scaling up" current AI approaches could lead to achieving artificial general...
By Craig S. Smith, Forbes | 03.08.2025
One recent evening in Shenzhen, a group of software engineers gathered in a dimly lit co-working space, furiously typing as they monitored the performance of a new AI system. The air was electric, thick with the hum of servers and...
