Today, Myogenica, a startup from the University of Minnesota, announced that the U.S. Food and Drug Administration has approved their Investigational New Drug (IND) application for MyoPAXon. This product, derived from induced pluripotent stem cells, is designed to regenerate skeletal muscle. The upcoming clinical trial will focus on assessing the safety, tolerability, and engraftment of MyoPAXon in patients with Duchenne muscular dystrophy (DMD).

Dr. Peter Kang, a pediatric neuromuscular neurologist and director of the Greg Marzolf Jr. Muscular Dystrophy Center at the University of Minnesota Medical School, will lead the early-stage clinical trial. The research team will administer MyoPAXon through intra-muscular injections to non-ambulatory adult DMD patients, monitoring for potential site reactions, immune responses, and the presence of dystrophin-producing myofibers.

"We believe stem cells have enormous potential for muscle regeneration in muscular dystrophy, and now is the perfect time to advance to human clinical trials for Duchenne muscular dystrophy," Kang stated.

If the initial trial shows positive results, the team plans to initiate a mid-stage study targeting additional muscles and evaluating muscle function. MyoPAXon's non-specificity to any genetic mutation means it could potentially treat a variety of muscular dystrophies and other muscle degenerative conditions.

This advancement is the result of years of interdisciplinary research at the University of Minnesota, involving the lab of Dr. Rita Perlingeiro, the Molecular and Cellular Therapeutics cGMP facility, the Center for Translational Medicine, and the Greg Marzolf Jr. Muscular Dystrophy Center. Duchenne UK has played a significant role, contributing over $900,000 to support the IND-enabling studies of MyoPAXon led by Perlingeiro. Additional funding was provided by the Department of Defense, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, MyDirectives, and the Greg Marzolf Jr. Foundation.

Myogenica, co-founded by Perlingeiro, holds exclusive licensing rights to the intellectual property behind MyoPAXon. Perlingeiro expressed her excitement, stating, "After many years of foundational and preclinical work, we are thrilled to have FDA approval for an IND application for an iPSC-derived myogenic cell product. This is a significant achievement, and we look forward to the future."

The University of Minnesota, which holds equity and stands to receive royalties under a license agreement with Myogenica, has reviewed and managed these interests according to its conflict of interest policies.

 

Source: prnewswire.com

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