Phase 3 findings could position the biotech’s therapy to become part of a “new standard” for SMA, its CEO said, while boosting research into whether it can preserve muscle in people with obesity.

The study, of Intellia’s treatment for hereditary angioedema, is the second Phase 3 trial the biotech has begun testing its “in vivo” gene editing medicines.

Producing safe, effective, high-quality viral vector therapeutics depends on a robust and tailored analytical testing strategy.

The Cambridge, Massachusetts-based biotech is using what it calls ligand-siRNA conjugates to reach drug targets in the kidney, which historically have been difficult to reach with RNA therapies.

Bristol Myers' immunotherapy can now be used before and after surgery in the U.S. Elsewhere, J&J, Bayer, Bristol Myers Squibb and Pfizer are trimming staff.

Having already been on a winding journey, Enjaymo, which Sanofi acquired through a 2018 buyout of Bioverativ, is now headed to Recordati as part of a deal announced Friday.

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The agency will offer earlier meetings with drugmakers as well as solicit more patient feedback on the process.

Today, a brief rundown of news from Metsera and Novartis, as well as updates from Sanofi and Wave Life Sciences that you may have missed.