Diogo Pinto,  —

Insilico Medicine and A2A Pharmaceuticals, two leading artificial intelligence (AI) biotechs, are collaborating to create a new company called Consortium.AI, which will advance AI for the development of new molecules to treat Duchenne muscular dystrophy (DMD) and other orphan diseases. Consortium.AI will feature computational new candidates designed…

Benitec Biopharma recently granted Axovant Sciences worldwide rights to its investigational AXO-AAV-OPMD gene therapy program for the treatment of oculopharyngeal muscular dystrophy (OPMD) under a global licensing agreement. AXO-AAV-OPMD is currently in preclinical development, and Axovant plans to launch a placebo-controlled trial testing the therapy candidate for OPMD…

The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) recommended expanding the use of Translarna (ataluren) to ambulatory children ages 2-5 with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation (nmDMD). CHMP’s recommendation of Translarna use in younger children is based…

Daiichi Sankyo announced positive safety findings in a Phase 1/2 trial of its investigational dystrophin gene therapy DS-5141 in a small group of Duchenne muscular dystrophy (DMD) patients, but noted results failed to show clear evidence of efficacy on a key endpoint. Evidence of exon-skipping was found, the company reported in…

Celebrex (celecoxib), an FDA-approved anti-inflammatory and pain reliever marketed by Pfizer, showed an ability to improve strength and function in leg, heart and diaphragm muscles of an animal model of Duchenne muscular dystrophy (DMD), a study found. Mice treated daily for one month with celecoxib also showed increases in…

Pfizer, Inc began a Phase 1b clinical trial (NCT03362502) for its investigational mini-dystrophin gene therapy product, PF-06939926, in patients with Duchenne muscular dystrophy (DMD). The first dose of the mini-dystrophin gene ( a shortened version of the human dystrophin gene) was given…

Omigapil, an investigational therapy by Santhera Pharmaceuticals, proved to be safe and well-tolerated in children and adolescents with congenital muscular dystrophy (CMD), in a Phase 1 clinical trial. Switzerland-based Santhera recently announced the successful completion of the CALLISTO trial (NCT01805024), which tested omigapil in pediatric patients with…