“CureDuchenne is a leader in funding pioneering research aimed at advancing the best possible therapies for DMD patients, and we value their vote of confidence and financial support of our snRNA-mediated exon skipping platform,” said Jim Burns, Ph.D., Locanabio’s chief executive officer.
My name is Debra Miller, founder and CEO of CureDuchenne, and the mother of an incredible son, Hawken, who has Duchenne muscular dystrophy. I’m here today to represent the voice of patient advocacy organizations serving those impacted by Duchenne – all of whom are in support of the accelerated approval of SRP-9001.
“CureDuchenne Ventures has a long history of investing in gene therapy and gene editing research to treat DMD, and MyoGene Bio’s approach is an excellent application of CRISPR/Cas9 technology to potentially address a large proportion – up to half – of DMD cases,” said Debra Miller, founder and chief executive officer of CureDuchenne. “
CureDuchenne Ventures announces an investment in Insmed Inc. to support the development of INS1201, their next generation intracerebroventricular (ICV) delivered AAV to directly address limitations of current gene therapy approaches.
See the below letter to the community
Join us for this pre-recorded presentation and Q&A discussing Dyne’s Phase 1/2 clinical trial, DELIVER, evaluating DYNE-251 for the treatment of individuals with Duchenne muscular dystrophy (DMD) who are amenable […]
As you may have read in the news, Sarepta Therapeutics today announced an update from the FDA on the review of SRP-9001, Sarepta’s experimental gene therapy for Duchenne muscular dystrophy. They shared the following information:
Dyne Therapeutics, who received early funding from CureDuchenne, has received FDA Orphan Drug and Rare Pediatric Designations for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy.
CureDuchenne Ventures Invests in hC Bioscience and its Novel tRNA-Based Therapeutic Approach for Duchenne Muscular Dystrophy