News

CRISPR-Cpf1 Corrects Duchenne Mutations, Restores Dystrophin Expression, UT Study Shows

Researchers from the University of Texas Southwestern Medical Center in Dallas have corrected Duchenne muscular dystrophy (DMD) mutations in human cells and experimental animal models using a new genome editing method called CRISPR-Cpf1. These new findings offer hope in permanently eliminating genetic mutations and rescue abnormalities linked to DMD. The study, “…

Bristol-Myers Squibb in Deals with Biogen, Roche to License Potential Therapies for Progressive Supranuclear Palsy, Duchenne

New York-based Bristol-Myers Squibb announced two separate agreements with Biogen of Cambridge, Mass., and Switzerland’s Roche to license compounds that aim to treat progressive supranuclear palsy (PSP) and Duchenne muscular dystrophy (DMD). Tau protein contributes to the stability of basic cellular structures known as microtubules to transport molecules. Abnormal addition of…

Catabasis Reports Some Setbacks in Clinical Trial of Edasalonexent (CAT-1004) for Duchenne

Catabasis Pharmaceuticals is facing challenges in its efforts to develop Edasalonexent (CAT-1004) — an oral drug that targets NF-kB, a protein activated in Duchenne muscular dystrophy (DMD) — to treat the rare degenerative disease. Edasalonexent is being evaluated in MoveDMD, an ongoing Phase 1/2 clinical trial (NCT02439216) consisting of…