Entos Pharmaceuticals Inc (Entos), a clinical-stage company that develops genetic medicines utilizing its non-viral, redosable Fusogenix PLV delivery platform is proud to announce a collaboration with the L-CMD Research Foundation, a nonprofit that urgently translates scientific research into treatments for LMNA-related congenital muscular dystrophy (L-CMD).

“We are very committed to developing gene therapies for rare and ultra-rare diseases like L-CMD with our Fusogenix PLV platform," said John Lewis, CEO of Entos Pharmaceuticals.

At least 200 children worldwide are diagnosed with L-CMD, a severe form of fatal muscular dystrophy characterized by progressive muscle weakness, heart arrhythmia and “dropped head”, and no treatments or cures. By combining Entos' Fusogenix PLV technology, that provides safe effective and redosable delivery of cargo, with novel gene editing approaches and muscle-targeted PLV formulations developed for our Duchenne Muscular Dystrophy program, the team will develop potentially curative therapies for this currently untreatable condition.

Entos is a clinical stage company with manufacturing and regulatory expertise in genetic medicines, and key infrastructure including a Good Manufacturing Practices (GMP) manufacturing facility in Carlsbad, and a planned R&D center and a GMP manufacturing facility in Edmonton, Alberta. This puts Entos in an ideal position to support in-house production of drug substances for IND-enabling studies and ultimately move curative therapeutics such as for LMNA-Related Congenital Muscular Dystrophy to the clinic.

“We are very committed to developing gene therapies for rare and ultra-rare diseases like L-CMD with our Fusogenix PLV platform," said John Lewis, CEO of Entos Pharmaceuticals. “The reality is that despite exciting advancements in gene editing technologies, traditional drug development pathways have failed for children with rare diseases and have left them and their families with no options. Collaborations like these can leverage emerging FDA pathways to make potentially curative genetic medicines accessible to those that really need them. We’re hopeful that our approach will make a real difference for the children with L-CMD and their families.”

"We are excited to partner with Entos Pharmaceuticals to advance redosable genetic therapy for L-CMD,” said Hannah Lowe, President of the L-CMD Research Foundation. “We are hopeful that with our combined experience, we can move swiftly to create a treatment pathway and platform for L-CMD therapies."