Andrew Joseph , 2025-05-05 08:30:00
MILAN — For more than three decades, a charity here has funded research aimed at developing medicines for genetic diseases. Recently, though, it added an unusual new role — as a gene therapy company of sorts.
The charity, the Telethon Foundation, took ownership from a small biotech of a drug that has been approved in Europe to treat an ultra-rare immune disorder called ADA-SCID. In taking such a step — something no nonprofit had done anywhere — it hoped to both rescue the gene therapy from disuse, and to come up with a new model for delivering these one-time, cutting-edge medicines in a financially sustainable way.
The fact that a charity — and not a biopharma company — now has the rights to the medicine is a reflection of the bleak situation the gene therapy field has found itself in, and how other groups, from nonprofits to academic researchers, are trying to mend the failures of the drug development system.
This article is exclusive to STAT+ subscribers
Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts.
Already have an account? Log in
To read the rest of this story subscribe to STAT+.
