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The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which is awaiting an FDA decision on or before Feb. 8.
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With the biopharma industry performing better of late, analysts, executives and other industry watchers are “cautiously optimistic”—a term heard all over the streets of San Francisco at the J.P. Morgan Healthcare Conference earlier this month.
Bristol Myers Squibb, GSK and Merck are contributing drug ingredients as part of their deals with the White House but are keeping many of the terms of their agreements private.
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.
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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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The BioSpace team is recording from San Francisco as they bring you the the latest highlights from Day 3 at JPM2024.
Triumvira Immunologics’ president and chief operating officer Rob Williamson noted that cell therapy companies might still have an uphill battle unless they can differentiate.
The Massachusetts–based startup will use the Series A funds to advance its pipeline of oral GPR17, CSF1R and TYK2 candidates.
Patients treated with AR-15512 showed higher rates of at least a 10-mm improvement in unanesthetized Schirmer’s score, a metric of tear production.
The BioSpace team is recording from San Francisco as they bring you the the latest highlights from Day 2 at JPM2024.
All assets of the regenerative medicine and cell therapy company are being divested to its research partner to the tune of $2 million in the form of a credit bid.
The Alliance for Regenerative Medicine predicts up to 17 cell and gene therapy approvals this year and defends high prices, while FDA’s Peter Marks expresses concern about manufacturing costs.
Following in the footsteps of Novartis, J&J and Merck, GSK jumped into the J.P. Morgan dealmaking frenzy Tuesday, picking up respiratory and inflammation-focused Aiolos Bio.
Vertex still has an ongoing diabetes partnership with CRISPR, which gives it a non-exclusive license to the latter’s platform to develop a potential cure for type 1 diabetes.
Following the FDA’s approval of the program, PhRMA is once again suing to block Florida from importing prescription drugs from Canada.