Moving away from a one-size-fits-all approach, the U.S. FDA's CBER released details Jan. 11 about how it’s leveraging its growing experience with cell and gene therapies (CGTs) to exercise greater regulatory flexibility in chemical, manufacturing and control requirements for the products.
Stoke Therapeutics Inc.’s speeded-up timeline for zorevunersen, the antisense oligonucleotide in development with Biogen Inc. as a first-in-class potential disease-modifying treatment for Dravet syndrome, put the rare, severe form of lifelong epilepsy in the spotlight. The news involved completion of enrollment and a phase III data readout from the Emperor study, as officials said signups of 150 patients are expected in the second quarter of the year, which puts the study on track for data in mid-2027.
It was a battle of the companies with drugs for transthyretin-mediated amyloidosis on the first day of the J.P. Morgan 2026 Healthcare Conference with Alnylam Inc., Bridgebio Pharma Inc. and Pfizer Inc. all presenting at the annual kickoff conference. One company disclosed 2025 sales as well as 2026 revenue guidance, the second only looked back, while the third was too big to do either for a specific drug.
With rumors regarding a couple of potential mega-mergers making the rounds, the week of the annual J.P. Morgan Healthcare Conference kicked off with the official disclosure of some billion-dollar collaborations, leading with Abbvie Inc.’s exclusive licensing deal with Remegen Co. Ltd. for PD-1/VEGF-targeted bispecific antibody RC-148.
Depending on who you ask, AI will take over the world and save it; or ruin it. Certainly, it is changing it. Science magazine dedicated its first editorial of 2026 to AI. Despite its title – “Resisting AI slop“ – editor-in-chief Holden Thorp gave the sort of nuanced review that is typical of him. “Like many tools, AI will allow the scientific community to do more if it picks the right ways to use it,” he wrote. “The community needs to be careful and not be swept up by the hype surrounding every AI product.”