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
After Merck noted the issue in its Q2 earnings call without providing specifics, analysts are left in the dark about the HPV vaccine’s future in China.
Lobbying groups and individuals connected with the industry are supporting candidates from both parties, with a particular focus on the future of the 340B discount program and pharmacy benefit managers.
Experts say the time is now to develop and provide widespread access to genetic medicines for the rarest diseases. What’s more, they say it is a moral imperative.
With U.S. election season now in full swing, BioSpace looks at pharmaceutical-associated campaign contributions. Plus, Q2 earnings, Adaptimmune’s big approval, an anticipated FDA decision on an MDMA-assisted treatment and more.
The size of the global immunology market is projected to nearly double by 2032 to a quarter of a trillion dollars. Here are this year’s standout deals in the space.
In this live panel discussion, speakers focused on the EU AI Act’s global regulatory implications for medical devices & QARA’s future. An executive summary is available.
In this live panel discussion, speakers navigate integrated ecosystems and the future of QARA. An executive summary is available.
In this live panel discussion, speakers explore artificial intelligence in medtech and practical realities in QARA. An executive summary is available.
After dropping $4 billion on a deal with the Japanese company last year, Merck is getting back some of its investment as Daiichi Sankyo buys into a Phase I/II T-cell engager therapy acquired from Harpoon Therapeutics.
Sangamo Therapeutics announced Tuesday it secured an exclusive licensing agreement with Roche’s Genentech, which is paying $50 million in near-term upfront fees and milestone payments to develop novel genomic medicines for neurodegenerative diseases.