Dyne Therapeutics Announces New Clinical Data from ACHIEVE Trial of DYNE-101 in DM1 and DELIVER Trial of DYNE-251 in DMD Demonstrating Compelling Impact on Key Disease Biomarkers and Improvement in Multiple Functional Endpoints

As early investors in Dyne Therapeutics, we are pleased to share they reported positive updates in their next-generation exon skipping program for Duchenne amenable to skipping exon 51.  In their phase 1/2 Trial, Dyne-251 at dose of 10 mg/kg given once a month showed an average 3.2% normal dystrophin expression (7.6% normal dystrophin when adjusted for muscle content) at 6 months.  Trends in functional improvements like the NSAA and other tests were observed, but non reached statistical significance at this time.  Dyne-251 has fully enrolled cohorts for 20 and 40 mg/kg dosing and is reporting favorable safety profiles, while dystrophin and functional read outs for these higher doses will be coming in the future. 

Dyne also reported positive safety, biomarker, functional, and patient reported outcome data in  Type 1 Myotonic Dystrophy program (DM1), and anticipates providing updates by the end of the year on their plans for pursuing expedited FDA approvals in both DM1 and Duchenne.

Read more from Dyne: https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-new-clinical-data-achieve-trial-dyne

Related Posts

Share This Page

Make an Impact

You can advance the care, treatment and cure for Duchenne muscular dystrophy. Contributions in any amount can truly make a difference and can be credited to a fundraiser or event from the list below.