Myofinity Biosciences Inc.

Myofinity Biosciences Inc. is a for-profit biotechnology company focused on developing novel protein therapeutics to treat rare muscular dystrophies. Through various prime federal grant awards from the National Institutes of Health, the company has advanced recombinant protein and gene editing technologies toward clinical trials for Duchenne muscular dystrophy, Becker muscular dystrophy, and limb-girdle muscular dystrophy type 2B.

Specifically, Myofinity Biosciences has received funding to optimize a TRIM72/MG53 protein therapeutic to enhance membrane repair in skeletal and cardiac muscle cells compromised in Becker muscular dystrophy. Additional NIH grants have supported the translational development and testing of a CRISPR gene editing approach (CRD-002) to remove duplication mutations underlying 10-15% of Duchenne cases. Myofinity has also received funding to engineer and test a recombinant human MG53 (rhMG53) protein called Myotrim to treat dysferlinopathies by enhancing membrane repair capacity. These projects have been funded through the NIH's National Institute of Arthritis and Musculoskeletal and Skin Diseases as well as the National Human Genome Research Institute, with the goal of generating preclinical data to support Investigational New Drug applications to the FDA for clinical evaluation. Through these awards, Myofinity Biosciences aims to advance mutation-targeted therapies for rare muscular dystrophies toward regulatory approval and clinical use.