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After a patient safety signal and then death, the FDA in October 2025 placed holds on two of the company’s CRISPR programs for hereditary transthyretin amyloidosis.
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Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.
From opening new therapeutic mechanisms to repairing neuronal damage, investigational molecules from Ventyx Therapeutics, AC Immune, Gain Therapeutics and more could shape the future of Parkinson’s disease treatment.
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The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
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Gene therapies have ridden investor mania to huge valuations but commercialization challenges have pushed market caps to the floor. At a roundtable last week, FDA leaders promised faster approvals and broad support to the industry.
BioSpace did a deep dive into executive pay, examining the highest compensation packages, pay ratios and golden parachutes—what a CEO would get paid to leave.
Gilead underscored its faith in the combo therapy and pledged to work with regulators to resolve the hold, which has paused five clinical trials. Gilead also stressed that the hold does not impact any other assets in its HIV pipeline.
Robert F. Kennedy Jr.’s removal of all remaining members of the CDC’s Advisory Committee on Immunization Practices raises questions about the upcoming meeting later this month. Analysts fear the committee could be more sympathetic to the HHS Secretary’s anti-vax viewpoints.
As of March 31, Recursion Pharmaceuticals had a cash position of $509 million. Following Tuesday’s layoffs, the biotech expects its runway to last into the fourth quarter of 2027.
Odyssey filed for an IPO in January but never revealed a fundraising target.
Merck’s Enflonsia will go up against Sanofi and AstraZeneca’s Beyfortus, which the partners plan to ship out early in the third quarter.
The reinstatement of the generic drug policy office is the latest reversal of course for Robert F. Kennedy Jr.'s HHS, which also recently rehired FDA staff responsible for making travel arrangements and those involved in user fee program negotiations.
Two weeks of upheaval at the CDC culminated Monday in the complete reconstitution of the CDC’s Advisory Committee on Immunization Practices as HHS Secretary Robert F. Kennedy Jr. pens op-ed criticizing “conflicts of interest” he says exist on the current committee.
Avidity has been given the go-ahead by the FDA to request accelerated approval of delpacibart braxlosiran—potentially the first disease-modifying treatment for facioscapulohumeral muscular dystrophy—with an application in the second half of 2026.