Pfizer’s phase 3 gene therapy trial has failed to demonstrate significant improvement in motor function for boys with Duchenne muscular dystrophy (DMD).

Invizius has completed a first-in-human study of its approach to taming the immune system’s overreaction to dialysis—with the goal of addressing the complications that can come with “angry blood.”

Abbvie, Merck, Roche, Genentech, Regeneron, Sanofi and other big names in biopharma have signed on to a new project from the Digital Medicine Society’s (DiMe) Digital Health Measurement Collaborative Community (DATAcc) that aims to set industry benchmarks for digital technologies.

GSK and Ochre Bio will work together in a $37.5 million partnership to pinpoint the drivers of liver disease.

Ultragenyx and Mereo BioPharma have broken off another piece of brittle bone data, linking their drug candidate to sustained reductions in fracture rates in a midphase clinical trial.

NodThera’s NLRP3 inhibitor has already proved its worth against Novo Nordisk’s blockbuster obesity drug in preclinical trials. Now, an early readout from a study in humans with obesity has again demonstrated the drug’s anti-inflammatory potential.

Avidity Biosciences’ AOC 1020, newly dubbed del-brax, demonstrated a safe profile and was tied to functional improvement among people with facioscapulohumeral muscular dystrophy (FSHD) in a phase 1/2 trial.

Lokavant has released a new artificial intelligence solution it claims can help contract research organizations and sponsors predict, optimize and control clinical trials in real time.

It remains to be seen whether Sage Therapeutics’ dalzanemdor can improve function in patients with Huntington’s disease. While a phase 2 trial has clarified the science around cognitive decline in patients with the neurodegenerative disorder, analysts have labeled the data on the drug itself as “underwhelming.”

Charles River Laboratories has launched a new program to come up with an animal-free alternative to inhalation toxicology tests, marking another move in its larger plan to reduce reliance on animal testing. Financial details were not disclosed.