As an early funder of Avidity Biosciences, we are pleased to share that AOC 1044, designed to skip exon 44 in the dystrophin gene, produced significant exon skipping in healthy volunteers.
– First participant is expected to be dosed in September 2023 with data anticipated in the second half of 2024 – – Cash runway extended through the end of 2025 […]
The first full day of FUTURES has wrapped! In the last couple of hours, we’ve had educational workshops on gene therapy, RNA-based therapeutics, and muscle restoration and anti-inflammatory treatments. Plus, everyone’s had a chance to meet a new person and create relationships that will last a lifetime. It’s been a lot to digest, so here’s the top highlights from Friday at CureDuchenne FUTURES.
CureDuchenne Ventures Invests in hC Bioscience and its Novel tRNA-Based Therapeutic Approach for Duchenne Muscular Dystrophy
Entrada Therapeutics announced the selection of a second clinical candidate, ENTR-601-45, for the potential treatment of people living with Duchenne who are exon 45 skipping amenable. Read more in the […]
Kevin Flanigan, MD is a leading expert in the development of gene therapies for Duchenne, and the director of the Center for Gene Therapy in the Abigail Wexner Research Institute […]
Entrada Therapeutics announced encouraging new data on their exon 44 skipping program following their presentation at TIDES USA 2022. CureDuchenne provided funding to Entrada last year as part of their Series […]
National Center of Neurology and Psychiatry and Nippon Shinyaku announce positive results for a Phase I/II study evaluating the efficacy and safety of their exon 44 skipping drug (NS-089/NCNP-02) Results […]
**UPDATE** Duchenne gene therapy developers and scientific experts discuss AAV in DMD: Antitransgene SAEs. Please watch this webinar to learn more about recent concerns in gene therapy clinical trials. **ORIGINAL POST** […]
