Dyne Therapeutics recently announced the publication of Duchenne muscular dystrophy (DMD) preclinical data in a publication titled, “Enhanced exon skipping and prolonged dystrophin restoration achieved by TfR1-targeted delivery of antisense oligonucleotide using FORCE conjugation in MDX mice” in Nucleic Acids Research.
The data published demonstrates that the FORCE™ platform achieved robust and durable dystrophin expression in multiple muscle tissues and significant improvement in muscle function in MDX mice, a well-established preclinical model of DMD. The article outlines in vivo data in the DMD MDX mouse model that demonstrated that a single dose of a mouse-specific FORCE conjugate (FORCE-M23D) enhanced muscle delivery leading to robust and durable dystrophin expression with up to 51% of wild-type levels in quadriceps, 72% in tibialis anterior, 62% in gastrocnemius, 90% in the diaphragm, and 77% in the heart.
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