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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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Despite not having a single candidate in the clinical stage, the Moderna-backed biotech is offering 6.25 million shares for $15 apiece in an initial public offering. Shares are expected to begin trading Friday.
The CEOs of BMS, J&J and Merck testified Thursday before the Senate health committee that pharmacy benefit managers bear much of the blame for high pricing, while declining to commit to price cuts.
Following promising Phase IIb studies, Takeda will advance its oral ORX2 agonist TAK-861 into Phase III studies for narcolepsy type 1, while nixing the candidate’s development in narcolepsy type 2.
Investors drove up the price of Kyverna Therapeutics’ stock by 59% in its initial public offering on Thursday afternoon, the first day of trading, reaching a peak per-share price of $35.01.
Gilead Sciences is ending the development of magrolimab for the treatment of blood cancer following the FDA’s placement of a clinical hold on all its programs related to the drug.
Kyowa Kirin’s dealmaking continued on Wednesday when BridgeBio Pharma granted the Japanese company an exclusive license to develop and commercialize infigratinib.
BioNTech will pay $50 million in cash and purchase $200 million of Autolus Therapeutics’ shares to progress the companies’ respective CAR-T candidates to commercialization.
On Thursday, Kyverna Therapeutics is debuting on the Nasdaq with an upsized initial public offering which the biotech will use to support its pipeline of anti-CD19 CAR T therapy candidates.
Thursday’s full-year financial results from AstraZeneca showed a revenue crash of more than 90% for COVID-19 medicines, while cancer, cardiovascular and rare diseases all generated double-digit revenue growth.
The European Medicines Agency is looking into how Novo Holdings’ recently announced $16.5 billion acquisition of contract manufacturer Catalent might affect the availability of drugs.