SATGENO: Regenerative gene repair therapy for muscular dystrophies

ongoing

Muscular dystrophies (MDs) are a group of about 50 different monogenic disorders characterized by progressive, irreversible muscle wasting, leading to loss of autonomy and premature death. Patients rely on palliative care; the disease burden is immense. Current gene therapy approaches do not address the underlying genetic causes. The challenges to develop gene therapies for MDs include the genetic variability and the lack of a safe drug delivery methods to muscle. MyoPax is a clinic-ready company focused on restoring muscle function in patients with muscle diseases via targeted regenerative therapies. For genetic MDs we have developed the Satgeno platform: We aim to harness the body’s natural regenerative capabilities to sustainably deliver a gene repair therapy for the first time to muscle. Satgeno leverages our patented stem cell technology to manufacture highly regenerative muscle stem (“satellite”) cells called Primary Human Satellite cells-derived muscle stem cells (PHSats). PHSats rebuild muscle tissue and, very importantly, generate new muscle stem cells in their respective niche. This capability allows us to 1) correct MD-causing mutations ex vivo in PHSats using virus-free, mRNA-mediated, CRISPR-derived gene editing, 2) deliver gene-corrected healthy stem cells to functionally important muscles in MD patients, and 3) sustainably rebuild healthy muscle function. With EIC support, we will conduct the first-in-human DELI550 trial, focusing on a frequent founder mutation. Pending safety and efficacy validation, we expand our approach into a comprehensive platform to treat various MD mutations affecting 400K+ people in the EU and US. This trial will mark a breakthrough. The platform has the potential to transform the patient´s treatment as acknowledged by Nature medicine “11 Clinical Trials that Will Shape Medicine in 2023”. We strive for a future where individuals with MDs experience significantly improved quality of life and functional independence.