Cartesian Therapeutics has claimed a phase 2b win for its BCMA-directed CAR-T therapy in generalized myasthenia gravis (gMG). But the biotech’s victory came on a primary endpoint it changed in May and in a population that excluded the 25% of participants treated at community clinics.

Mouse models of GBA1 Parkinson’s disease that are treated with Gain Therapeutics’ clinical-stage drug appear to have better cognitive function and lower disease biomarkers than untreated animals.

Issues with ventilators across three major manufacturers—Philips, Baxter and Zoll—have led to a string of Class I recall notices from the FDA, the agency’s most severe level.

Merck & Co. is expanding a research partnership with Orion into an exclusive licensing pact, putting more than $1.6 billion biobucks on the table for cancer candidates targeting CYP11A1, an enzyme important in steroid production.

Dexcom is laying off more than 500 employees at its San Diego location, while it looks to expand its manufacturing operations elsewhere after growing too big for its Southern California home.

A new gene editing technique derived from bacterial “jumping genes” can add, remove, recombine and invert DNA sequences, potentially overcoming some of the limitations of CRISPR.

Ensho Therapeutics has emerged with an inflammatory disease therapy portfolio from Eisai’s GI subsidiary EA Pharma, including a phase 2-ready clinical program in ulcerative colitis (UC).

Rocket Pharmaceuticals’ flight path to approval for Kresladi has been delayed again after the FDA issued a complete response letter (CRL) requesting additional manufacturing information.

The FDA has launched a project with the Bill and Melinda Gates Foundation to help aid the development of breath-based diagnostics for detecting diseases more easily among underserved populations, including in rural, remote and impoverished areas.

Ethris’ mRNA-based treatment to prevent viral infections in people with uncontrolled asthma so far appears to be safe and increases expression of its target protein, early data suggest.