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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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The FDA’s busy week ahead involves three decision dates for potential industry firsts and a highly anticipated advisory committee meeting for two CAR-T therapies.
Novo Nordisk’s early-stage amylin and GLP-1 co-agonist elicited a 13.1% reduction in body weight, with an overall favorable safety profile, the Danish drugmaker reported Thursday at an investor event.
The Anglo-Swedish drugmaker is making the investment to bolster its vaccine manufacturing and research and development capabilities in the U.K.
President Joe Biden has proposed expansions to the Inflation Reduction Act, including an increase in the annual number of prescription drugs that would be subject to price negotiations to 50, up from 20.
The early-stage oncology startup filed for an initial public offering on Wednesday, seeking funds to help develop its novel cancer therapies targeting extrachromosomal DNA.
The recently launched company shared two posters at the Muscular Dystrophy Association’s annual conference this week ahead of a March 21 PDUFA date.
Presentations are standard requirements in the hiring process for some biopharma positions. Here’s how to approach them.
A mid-stage study of Alnylam Therapeutics’ experimental RNAi-based drug to treat hypertension met its primary endpoint.
Separate challenges exist for companies developing gene therapies for rare and common cardiovascular conditions, experts told BioSpace.
Sionna Therapeutics on Wednesday announced it raised another round of funding as the company looks to challenge Vertex Pharmaceuticals’ dominant cystic fibrosis business.