A bespoke CRISPR therapy suggests a blueprint for treating ‘N-of-1’ diseases A gene editing drug custom-made for a critically ill baby showed that, for some ultra-rare diseases, it’s possible to design and test a new CRISPR medicine in just a few months.

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 The sweeping workforce reduction that began Tuesday was made worse by mistakes and poor communication, including directions for some CMS employees to contact a director who died last year.

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

Cell, gene therapy makers lose a champion at FDA with exit of Peter Marks Marks’ resignation leaves the field without a regulator many view as “integral” to its progress over the last decade.

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...

First Prader-Willi drug to reduce hunger approved by FDA Clearance of Vykat unlocks what analysts anticipate will be a lucrative market opportunity for Soleno Therapeutics.

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 drug gets long-awaited FDA approval in deadly heart disease Amvuttra’s clearance in a rare form of cardiomyopathy could help Alnylam turn a regular profit — if it can wrest control of a competitive and fast-changing market.

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 gets first coverage nod from major insurer Optum, the pharmacy benefit manager owned by UnitedHealth Group, placed Journavx on some of its commercial formularies in an interim decision while a formal review continues.

Vertex’s new pain drug gains interim coverage from UnitedHealth's Optum:

www.biopharmadive.com/news/vertex-...

Blocking PD-1 and VEGF: The bispecific cancer drugs that could best Keytruda Striking study results last year indicated a new type of medicine may improve on Merck’s dominant immunotherapy, spurring a wave of research practically overnight.

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...