News
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.
FEATURED STORIES
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.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
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.
THE LATEST
AstraZeneca’s blockbuster BTK inhibitor Calquence significantly improved progression-free survival when used as a frontline treatment in patients with mantle cell lymphoma, according to Thursday’s late-stage results.
Following strong Phase II data, Amgen is going all in on its next-generation obesity treatment MariTide, with plans to run a Phase III trial and a separate mid-stage study for diabetes.
The race is on to develop therapeutic cancer vaccines that could give immunotherapies an edge, and late-stage trials could soon provide more-robust data about candidates’ efficacy and safety.
The new company, BridgeBio Oncology Therapeutics, is looking to advance two KRAS inhibitors and a blocker of the interaction between the RAS and PI3K pathways.
The Swiss drugmaker is paying $1 billion and committing up to $750 million in milestones for Mariana Oncology’s preclinical cancer pipeline and clinical supply capabilities, the companies announced Thursday.
Regeneron Pharmaceuticals on Thursday reported $1.4 billion in sales of its blockbuster eye drug in the first quarter of 2024, falling short of Wall Street’s expectations of $1.86 billion.
Novo Nordisk on Thursday reported a jump in first-quarter 2024 revenue, driven by a more than 100% increase in sales of weight-loss drug Wegovy and the strong performance of type 2 diabetes medication Ozempic.
Boehringer Ingelheim on Wednesday won the FDA’s approval for a high-concentration and citrate-free version of its Humira biosimilar Cyltezo, targeting AbbVie’s blockbuster drug.
Citing a strategic prioritization by Moderna, Metagenomi announced Wednesday that the partners have “mutually agreed” to terminate their gene editing agreement.
Astellas Pharma and Poseida Therapeutics have entered into a second CAR-T contract to develop novel and flexible allogeneic cell therapies in oncology.