ABOUT THE FORCE PLATFORM
Dyne Therapeutics, Inc., a muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, announced new data regarding its Duchenne muscular dystrophy (DMD) program that demonstrate robust and durable exon skipping and dystrophin expression in both cardiac and skeletal muscles in in vivo models.
The most recent data were generated using the mdx mouse model, a validated DMD disease model which has a mutation in exon 23. In this model, Dyne’s FORCE platform achieved:
- Robust, durable exon skipping in cardiac and skeletal muscles after a single dose
- Substantial dystrophin expression after a single dose of 30 mg/kg at 4 weeks:
- 90% of wild-type dystrophin restoration by Western Blot with approximately 80% dystrophin-positive fibers in the diaphragm
- 78% of wild-type dystrophin restoration by Western Blot with approximately 80% dystrophin-positive fibers in the heart
- 46% of wild-type dystrophin restoration by Western Blot with approximately 68% dystrophin-positive fibers in the quadriceps
- Durable expression and dystrophin-positive fibers detected out to 12 weeks after single dose
These new data build on previous results in the mdx model showing treatment with FORCE resulted in enhanced functional benefit in multiple standardized assessments and a reduction in serum creatinine kinase, a biomarker of muscle damage.
In non-human primate studies, DYNE-251 demonstrated:
- Robust exon 51 skipping in cardiac and skeletal muscles after 5 weekly 30 mg/kg doses measured 8 weeks post-initial dose:
- 52% exon skipping in the diaphragm; 43% exon skipping in the heart; 18% exon skipping in the quadriceps
- A favorable safety profile in a GLP toxicology study with no dose-limiting toxicity observed after 5 weekly doses up to a maximally feasible dose, thus supporting advancement into the clinic.
The presentation, “FORCE™ platform delivers exon skipping PMO, leads to durable increases in dystrophin protein in mdx mice and is well tolerated in NHPs” is available in the Scientific Publications & Presentations section of Dyne’s website.
ABOUT DYNE THERAPEUTICS
Dyne Therapeutics is building a leading muscle disease company dedicated to advancing innovative life-transforming therapeutics for people living with genetically driven diseases. With its proprietary FORCE™ platform, Dyne is developing modern oligonucleotide therapeutics that are designed to overcome limitations in delivery to muscle tissue seen with other approaches. Dyne’s broad portfolio of therapeutic candidates for serious muscle diseases includes programs for myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD).
For more information, please visit https://www.dyne-tx.com/.