New: A #CRISPR gene editing drug custom-made for a critically ill baby in just a few months appeared to be safe and working as intended, according to results newly published in the New England Journal of Medicine. Details below: #biotech #biosky #ASGCT2025 www.biopharmadive.com/news/crispr-...

‘A cruel April Fool’s joke’: HHS layoffs characterized by confusion, errors www.biopharmadive.com/news/hhs-lay... via Healthcare Dive's Rebecca Pifer.

New: The resignation of Peter Marks leaves the cell and gene therapy field without a regulator many view as “integral” to its progress over the last decade. #biosky #biotech #genetherapy www.biopharmadive.com/news/peter-m...

FDA approves Soleno Therapeutics drug to treat the intense hunger associated with Prader-Willi syndrome. The OK is a milestone for research into the rare disease, which has frustrated drugmakers' prior attempts. More from @benthefidler.bsky.social here: www.biopharmadive.com/news/soleno-...

Alnylam gets expanded FDA approval for Amvuttra, which is now cleared to treat transthyretin amyloidosis with cardiomyopathy. The new indication is seen as vital for Alnylam's growth, as @benthefidler.bsky.social reports: www.biopharmadive.com/news/alnylam...

Vertex’s new pain drug gains interim coverage from UnitedHealth's Optum: www.biopharmadive.com/news/vertex-...

New: an in-depth look at the fast-growing effort to determine whether an emerging class of multi-pronged, PD-1 x VEGF-targeting drugs can build upon Keytruda and other cancer immunotherapies like it. #biosky #biotech www.biopharmadive.com/news/pd1-veg...