DYNE-251 for DMD, DYNE-101 for DM1 showing safety in early trials

DYNE-251, an experimental treatment for Duchenne muscular dystrophy (DMD), is being well tolerated by patients in an ongoing clinical trial, according to the therapy’s developer, Dyne Therapeutics. Dyne also reported positive safety data from an ongoing trial of DYNE-101, the company’s investigational therapy for myotonic dystrophy type…

FDA grants orphan drug, rare pediatric status for DYNE-251

DYNE-251, an investigational therapy for Duchenne muscular dystrophy (DMD), has received orphan drug and rare pediatric disease designations from the U.S. Food and Drug Administration (FDA). Orphan drug status is meant to support the development of therapies for rare conditions affecting less than 200,000 people in the U.S. It…

DMD Treatment Candidate DYNE-251 Granted FDA Fast Track Status

The U.S. Food and Drug Administration (FDA) has granted fast track designation to Dyne Therapeutics’ DYNE-251 for the treatment of people with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 51 skipping. Fast track status is intended to accelerate a therapy’s development and expedite…

First Patient Has Received DYNE-251 in DELIVER Trial, Dyne Announces

The first patient has received Dyne Therapeutics‘ investigational therapy DYNE-251 for treating Duchenne muscular dystrophy (DMD), the company has announced. The therapy is being tested in the DELIVER Phase 1/2 clinical trial and is indicated for patients with mutations amenable to exon 51 skipping. “We are excited to…

FORCE Platform Delivers Exon-skipping Therapy Directly to Muscles

Dyne Therapeutics’ FORCE platform delivered its investigational exon-skipping therapy for Duchenne muscular dystrophy (DMD) directly to muscles in a mouse model of the disease, according to the results of a preclinical study. The potential DMD treatment improved dystrophin protein production and enhanced physical activity in treated mice. The…

FDA Places Hold on DYNE-251 Clinical Trial Launch in DMD

The U.S. Food and Drug Administration (FDA) has placed a hold on Dyne Therapeutics’ application to launch a clinical trial evaluating DYNE-251, an experimental exon-skipping therapy for Duchenne muscular dystrophy (DMD). Dyne now hopes to have that Phase 1/2 trial underway by June, the company said in a …