News
The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which is awaiting an FDA decision on or before Feb. 8.
FEATURED STORIES
With the biopharma industry performing better of late, analysts, executives and other industry watchers are “cautiously optimistic”—a term heard all over the streets of San Francisco at the J.P. Morgan Healthcare Conference earlier this month.
Bristol Myers Squibb, GSK and Merck are contributing drug ingredients as part of their deals with the White House but are keeping many of the terms of their agreements private.
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.
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
The partnership will allow BMS to advance a T cell–based therapy that is only activated once in the vicinity of a tumor.
The U.S. regulator shared the roadmap for implementing the program, first proposed in August 2025, and teased changes made in response to industry feedback.
The company also stands to gain from recent regulatory FDA guidance aimed at streamlining the development of non-opioid painkillers, Jefferies analysts suggested.
Pfizer CEO Albert Bourla said that the main thing getting in the way of changing vaccine discussions in the U.S. is the Secretary of Health, Robert F. Kennedy, Jr.
In this episode of Denatured, BioSpace editorial team members, Senior Editor, Annalee Armstrong, and News Editor, Dan Samorodnitsky, discuss their post-JPM takeaways and 2026 forecasts after speaking to a range of pharma and biotech executives and investors last week.
Fueled by advances in biomarkers, brain mapping, and AI, investment in neuro and CNS innovation shows no signs of slowing, even amid costly setbacks. BioSpace spoke to investors at JPM26 to get their take on recent bets in new therapies and neurotech.
Reporting Q4 and full year earnings on Wednesday, J&J executives hailed growth across the healthcare giant’s portfolio while standing fast on its talc lawsuit and tariffs.
Attendance at the Biotech CEO Sisterhood’s annual photo of women leaders and allies in Union Square doubled this year. There’s still more work to do.
After winning a surprise approval for its hereditary angioedema drug Ekterly, KalVista is confident the oral offering will capture the lion’s share of the market for on-demand use.
Novo Nordisk pulled back from cell therapies last October, scrapping development of a type 1 diabetes therapy and laying off most employees working on this modality.