In December 2020, CureDuchenne Ventures announced funding support for PepGen, Inc. to advance their Duchenne exon-skipping program to the clinic for trial. PepGen’s EDO cell-penetrating peptide platform is designed to improve the delivery of […]
Solid Biosciences issued a press release providing new, positive 2-year data from their IGNITE DMD clinical study in advance of the 2022 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. […]
We’re thrilled to share news about Code Biotherapeutics, a company pioneering targeted non-viral delivery of genetic medicines. CureDuchenne’s April 2021 research investment into Code Biotherapeutics was based on extensive preclinical […]
In 2015, CureDuchenne supplied early funding to Capricor Therapeutics in support of a novel, first-in-class therapy to improve cardiac function in people with Duchenne muscular dystrophy. Duchenne can cause debilitating […]
On January 14th 2022, the U.S. FDA placed a clinical hold on Dyne’s Investigational New Drug (IND) submission for the launch of their clinical trial of DYNE-251, which targets Duchenne […]
Solid Biosciences provided an update on their programs this week. Notably, their microdystrophin Phase 1/2 trial continues to dose participants, and they are on track to report dystrophin levels and […]
Sarepta today announced topline results from Part 2 of Study 102, an ongoing, randomized, double-blind, placebo-controlled clinical trial evaluating its investigational gene transfer therapy SRP-9001 in Duchenne patients. In […]
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 in Participants With Duchenne Muscular Dystrophy Sarepta is now expanding their trial of SRP-9001 (ENDEAVOR) to […]
We would like to share a recent change in our gene therapy clinical program for DMD. Three serious adverse events of muscle weakness, two of which involved myocarditis (inflammation of […]
