NanoCas, a smaller version of CRISPR tested with a single AAV, delivers on-target results

Mammoth Biosciences researchers have developed NanoCas, an ultracompact CRISPR nuclease, demonstrating its ability to perform ...
Morningstar

Dyno Therapeutics Unveils Best-in-Class Dyno-bn8 AAV Vector for Muscle Gene Therapies, Achieving Therapeutic Delivery With Improved Safety Potential

Non-human primate study results demonstrate improvements in skeletal and cardiac muscle delivery efficacy, safety potential, human translatability, and manufacturability over prior AAV capsids ...
KXAN

Guidelines to standardize care, enhance safety monitoring, and promote equitable access to transformative treatments.

New York, Aug. 23, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy (PPMD) today announced the publication of jointly developed consensus ...
Seeking Alpha

Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients Demonstrating Promise of the FORCE™ Platform ...

- All Evaluable Patients in the 3.4 mg/kg Cohort Treated with DYNE-101 Q4W Demonstrated Consistent Splicing Correction with a 19% Mean Improvement Across 22-Gene Panel at 3 Months - - Improvement in ...
Nasdaq

Wave Life Sciences Receives FDA Rare Pediatric Disease Designation for WVE-N531 for the Treatment of Duchenne Muscular Dystrophy

Designation highlights significant unmet needs in DMD; dystrophin data from potentially registrational FORWARD-53 trial of WVE-N531 are on track for 3Q 2024 In previous Part A trial, WVE-N531 achieved ...
Nanowerk

Delivering genome editing components with Lipid Nanoparticles to repair the building blocks of muscles

Scientists have developed a novel approach of genome editing to repair muscle stem (satellite) cells, offering new hope for Duchenne muscular dystrophy (DMD) treatment. While adeno-associated virus ...