Clinical Translation of hESC-derived protein therapy that positively regulates the regenerative capacity of post-natal muscle for treating DM1 

With the support of a TRAN1 grant from CIRM, Juvena Therapeutics, Inc., progressed the clinical translation of a human embryonic stem cell-derived protein therapy that positively regulates the regenerative capacity of post-natal muscle for treating muscle diseases, such as myotonic dystrophy type 1 (DM1). DM1 is the most common muscular dystrophy in adults, affecting an estimated 1 in 2,532 people in the CA, slowly depriving patients of their ability to walk, use their hands, and breathe - and yet this population is without treatment options.   As part of grant-supported activities, Juvena developed a Good Manufacturing Process (GMP) compatible process and performed non-GMP production of a material supply of this protein therapy, an engineered version of a stem cell-secreted signaling protein, named JUV-161. To support the creation and use of this material, qualification of assays for the manufacturing process, release potency, and pre-clinical studies were conducted. Using this material Juvena demonstrated treatment with JUV-161 improves stem/precursor survival and differentiation, counters muscle atrophy, and improves muscle strength/endurance in multiple disease models, including DM1. Juvena performed pre-clinical toxicology, biodistribution, safety, and dose evaluation studies to support the clinical translation of a safe and effective dose of JUV-161 to humans. This work culminated in a positive early (pre-IND) meeting with the FDA where Juvena received clear direction and anticipated guidance on the proposed approach for advancing this new drug toward human clinical trial activities.