Can This Gene Therapy's 110% Dystrophin Expression Transform Duchenne Treatment? Key Trial Results Inside

CHMP recommends approving Vyjuvek for DEB patients from birth

A committee of the European Medicines Agency is recommending approving Vyjuvek (beremagene geperpavec), a gene therapy from Krystal Biotech, to treat wounds in patients of all ages with dystrophic epidermolysis bullosa (DEB). The opinion by the Committee for Medicinal Products for Human Use (CHMP) will now go to the European Commission for a final decision on marketing authorization. That’s expected in the second quarter of this year and will ap…

GenAssist Ltd Announces FDA Clearance of Investigational New Drug Application for GEN6050X, a Globally First-in-Class Base Editing Drug for Duchenne Muscular Dystrophy - Pipelinereview

SUZHOU, China I March 6, 2025 I On March 06, 2025, GenAssist Ltd (GenAssist), a pioneering gene-editing biotechnology company specializing in genome medicines, is thrilled to announce that it has received clearance from the U.S Food and Drug Administration(FDA) for its Investigational New Drug (IND) application for GEN6050X, a first-in-class base editing drug for Duchenne Muscular Dystrophy (DMD). GenAssist is planning to conduct clinical study …

Can This Gene Therapy's 110% Dystrophin Expression Transform Duchenne Treatment? Key Trial Results Inside

Promising DUCHENNE trial data shows 110% microdystrophin expression. FDA meeting planned for accelerated approval as $200M offering extends runway to 2027.

Freedom Through Accessible Transportation: Massachusetts Family Gets New Set of Wheels Through Jett Giving Fund - Jett Foundation

The Dinh family’s world felt cracked – slipping away by the day – years ago after learning that their two sons, Steven and Simon, were diagnosed with Duchenne muscular dystrophy, a progressive neuromuscular disorder causing a lack of motor, pulmonary, and cardiac function. Their parents, Kristina and Si, no longer imagined their boys’ childhood full of running, jumping, and playing without limits. Instead, they faced unforeseen challenges while …

Elevidys Could Become Transformational Gene Therapy for Duchenne Muscular Dystrophy

Elevidys offers significantly higher dystrophin production than existing Duchenne muscular dystrophy treatments but requires intensive monitoring for adverse events.