We at CureDuchenne are excited to acknowledge the recent announcement by the United States Food and Drug Administration (FDA) about the creation of the “Rare Disease Innovation Hub.” This new […]
REGENXBIO announced they are expanding their AFFINITY DUCHENNE Trial, which delivers a micro-dystrophin via AAV8, to include a new cohort of younger patients. The trial, which has been enrolling ambulatory […]
PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping
Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients
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 […]
