The company expects the trial to support the submission of a biologics license application with the US Food and Drug Administration in 2026.

Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for ...

A small trial of an experimental vaccine has shown promising results for patients with the most aggressive type of breast ...

RegenxBio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy ...

Specifically, StitchR restored the protein Dystrophin, which is absent in patients with Duchenne muscular dystrophy, and the ...

Regenxbio’s investigational gene therapy improved muscle function for patients with Duchenne muscular dystrophy (DMD) in a phase 1/2 study. | Regenxbio’s investigational gene therapy improved muscle ...

Regenxbio shares surged 20% after disclosing new, positive efficacy and safety data of its Duchenne muscular dystrophy treatment. Shares were recently trading around $11.55. The stock is down 46% on ...

Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...

a potential best-in-class gene therapy for Duchenne muscular dystrophy, has advanced to pivotal stage and dosed its first patient. The company also announced new, positive efficacy and safety data ...

Put up against placebo in the phase 3 EMBARK trial, delandistrogene moxeparvovec (Elevidys) did not significantly improve ...

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

In-shoe sensors, AI and biomarkers will monitor muscular dystrophy and atrophy in children, adolescents and adults — with ...