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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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Anticipating approval for its COPD therapy ensifentrine, Verona has entered into a $650 million financing deal with Oaktree Capital Management and OMERS Life Sciences.
With its $525 million investment, Royalty Pharma will acquire the royalties and milestones for ImmuNext’s anti-CD40 therapy frexalimab, which is currently in Phase III trials for multiple sclerosis.
With 15 patients started across its three gene therapies, bluebird bio claims a 138% year-over-year revenue growth and aims to initiate up to around 100 new patients in the current year.
At a Thursday ASGCT 2024 session, CBER Director Peter Marks made the case for a better, “more convergent” global framework on cell and gene therapies, especially for rare diseases.
The Bay Area–based biotech plans to use the funds to advance two lead programs, one to treat atopic dermatitis and another for immune-mediated diseases.
As competition with Eli Lilly heats up, Novo Nordisk has partnered with Flagship’s Metaphore Biotechnologies to take a biomimicry approach to GLP-1s.
After a combination with Eisai’s Lenvima failed to improve survival in advanced disease in December, adjuvant Keytruda plus chemotherapy missed the mark in newly diagnosed, high-risk patients.
An autologous and personalized regulatory T cell therapy is safe in patients with type 1 diabetes, but does not help preserve β-cell function.
Takeda saw a 57% drop in profit in its 2023 fiscal year and has lowered its outlook for the coming fiscal year, mostly driven by increasing generic competition for its ADHD treatment Vyvanse.
Launched in 2021, the public-private consortium on Wednesday updated ASGCT attendees on its efforts to bring adeno-associated virus gene therapies to more rare disease patients.