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
Blackstone joins other big investors such as ARCH Venture Partners and Bain Capital Life Sciences in pumping billions of dollars into the industry.
The greenlight for Journavx (suzetrigine), which comes on the heels of a $7.4 billion opioid settlement, could spark momentum in the fledgling non-opioid pain space.
Analysts were unfazed by the news that Takeda will cease development of soticlestat after Phase III failures, while responding positively to the announcement that Julie Kim will take the helm of the Japanese giant in 2026.
CAR T–focused biotech Cargo Therapeutics surprised and disappointed analysts when it announced that it would discontinue a mid-stage trial of its lead program, firi-cel.
Sanofi’s jump in earnings comes with an increased emphasis on R&D and vaccines, plus an eye cast toward M&A to shore up its pipeline.
For 2025, Roche will continue a careful approach to high-priced deals, putting science at the center of its business development decisions, executives said Thursday.
In this episode, presented by the Genscript Biotech Global Forum 2025, BioSpace’s Head of Insights Lori Ellis talks to Tom Whitehead, co-founder of the Emily Whitehead Foundation, about how standard care, cell and gene therapies and their impact on patients.
While the Chicago metropolitan area is not a major life sciences hub, a recent Cushman & Wakefield report predicts the Chicago market should be a growth spot in the coming years. Chicago Biomedical Consortium and COUR Pharmaceuticals executives share what makes the area a hot spot.
The layoffs will allow Ironwood to dedicate more resources to pushing its lead molecule apraglutide through a Phase III trial and a rolling NDA submission.
Kennedy’s confirmation hearing on Wednesday became heated as Democratic senators grilled the nominee for HHS Secretary on his previous statements about vaccine safety.