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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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Backed by ARCH Venture Partners, F-Prime Capital and Mubadala Capital, the new company will develop a pipeline of brain-penetrant small molecules to address inflammation, metabolic dysfunction and restoring lysosomal function.
In a deal expected to close in Q1 2025, Roche will gain access to Poseida’s off-the-shelf CAR T candidates.
By mid-2025, the biotech will split into two entities: a new, as-yet-unnamed innovative medicines specialist and a cell therapy company, the latter of which will inherit the Galapagos name.
The FDA recommended maintaining a minimum of 5% weight-loss for drug developers seeking to establish the efficacy of their investigational obesity candidates.
Maze’s IPO comes on the heels of its oversubscribed Series D funding round, which infused the biotech with $115 million in funding to support the development of its kidney disease pipeline.
A post-marketing review by the FDA detected an increased risk of the autoimmune condition in patients inoculated with GSK’s Arexvy and Pfizer’s Abrysvo, prompting the regulator to require adjustments to the vaccines’ labels.
The investment continues a Novo dealmaking spree to cement its leadership status in the cardiometabolic space, with partnerships with Photys Therapeutics, Ascendis Pharma and two Flagship-backed start-ups.
BioSpace presents 25 noteworthy biopharma startups in ’25; analysts forecast stronger M&A as the J.P. Morgan Healthcare Conference kicks off next week; GLP-1s continue to expand their reach as Novo, Lilly fight against compounders; and a look ahead to five key FDA decisions in Q1.
Licensing deals have risen in prominence in a restrained market environment. Is it desperation, or an important part of the biotech ecosystem? Experts weigh in.
Look for renewed investment driven by lower interest rates in the new year, and a continued focus on late-stage assets, oncology and reaping the benefits of AI.