The need-to-know this morning
Genetic medicine
How a charity intervened in a bid to save gene therapy
For decades, a small foundation in Milan quietly funded genetic research — but more recently, in an unprecedented turn, it has taken full ownership of a gene therapy after it was abandoned by biopharma companies that deemed it financially unsustainable. The charity, called the Telethon Foundation, is now navigating the complex world of regulatory filings, pricing negotiations, and manufacturing — all in an effort to rescue a treatment for an ultra-rare immune disorder called ADA-SCID.
Industry has been retreating from costly one-time therapies for rare diseases, so Telethon's experiment is being closely watched as a possible model for how academic institutions and nonprofits might step in when market forces fail patients.
"We need to show the world that this is doable," Celeste Scotti, head of research and development at the foundation, told STAT's Andrew Joseph.
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REGULATION
FDA will ask outside experts to review Capricor's therapy for Duchenne muscular dystrophy
From STAT's Adam Feuerstein: The FDA informed Capricor Therapeutics that a meeting of outside experts will be convened to review the company's experimental cell therapy designed to improve heart function in patients with Duchenne muscular dystrophy, the company said this morning.
An exact date for the FDA meeting has not yet been set, Capricor added.
The FDA's intention to convene a so-called advisory committee meeting so that outside experts can opine on the Capricor therapy called deramiocel adds risks to the ongoing review. It's also unsurprising given the novel nature of the therapy, which is derived from donor stromal cells, and the less-than-pristine clinical data submitted to the agency.
The FDA has sharply cut back on the number of advisory committee meetings it holds to review drug applications. The trend started during the tenure of Commissioner Robert Califf and has continued under current Commissioner Marty Makary.
The Capricor meeting is a signal that the FDA's core drug-review functions remain intact despite recent staffing cuts.
RESEARCH
Overseas grants could be 'closed down' by NIH under new policy
Last week, the NIH announced that it would freeze all "foreign subawards" — that is, agency funds that a U.S. researcher gives to collaborators in other countries. It's now reviewing its entire international research portfolio, STAT's Megan Molteni reports exclusively. Internal emails obtained by STAT reveal the agency's new mandate: Unless research abroad is irreplaceable and directly benefits Americans, it will be shut down.
The decision threatens thousands of studies on diseases like HIV, Alzheimer's, and rare genetic disorders that rely on international collaboration. The news prompted an outcry from researchers who warn the abrupt shift could cause lasting damage to biomedical progress.
"If you can't clearly justify why you are doing something overseas, as in it can't possibly be done anywhere else and it benefits the American people, then the project should be closed down," the email, which was sent by the NIH's principal deputy director, Matthew Memoli.
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