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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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Mature biopharma deals are stealing all the headlines, but Bristol Myers Squibb’s Robert Plenge says the company’s deals with insitro, Orbital and more are building the future.
The deal, which sees AbbVie paying RemeGen $650 million upfront, gives the pharma ex-China rights to the biotech’s PD-1/VEGF bispecific antibody—a modality being targeted by companies including BMS, Merck and Pfizer.
While Moderna’s full-year sales landed in the upper end of its target range, Jefferies analysts said further reductions are needed if the biotech hopes to hit its 2028 break-even target.
The deal will see Novartis gain global rights over SciNeuro’s potentially disease-modifying anti-amyloid antibody, which leverages the latter’s proprietary shuttle platform to allow delivery into the brain.
FDA Commissioner Marty Makary called these changes “common-sense reforms” that could expedite the development of cell and gene therapies.
Aurora joins the clutch of companies linked to Nobel Prize winner and CRISPR trailblazer Jennifer Doudna.
Hair loss–focused Veradermics and cancer biotech Eikon follow the lead of Aktis Oncology, which last week announced a $318 million IPO target.
In his annual letter, Flagship Pioneering’s Noubar Afeyan lays out a choice between near-term “human-made miracles” and a reversion to the pain and suffering of past diseases due to “growing contempt” in the U.S. for the scientific method.
Heightened diligence standards and longer decision timelines for early-stage startups slowed venture activity last year, J.P. Morgan found in a report published ahead of the bank’s annual healthcare conference in San Francisco.
The prevalence of serious inflammatory safety issues such as cytokine release syndrome and immune effector cell–associated neurotoxicity syndrome limits the reach of these transformative cancer therapies.