Entos Pharmaceuticals and the L-CMD Research Foundation Collaborate to Develop Curative Therapy for LMNA-Related Congenital Muscular Dystrophy

EDMONTON, Alberta & SAN DIEGO--(BUSINESS WIRE)--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).

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."

About L-CMD and the Research Foundation

L-CMD Research Foundation is a nonprofit formed in 2020 to urgently translate scientific research into treatments for LMNA-related congenital muscular dystrophy (L-CMD). The Houston-based nonprofit was created by the Corman family, Mark Corman and Hannah Lowe, after their younger son, Austin, was diagnosed with L-CMD when he was five months old. L-CMD is a severe form of muscular dystrophy characterized by progressive muscle weakness, heart arrhythmia and “dropped head;” it is thought to affect at least 200 children worldwide. Knowing that Austin’s condition would progressively worsen, leading to death, and that there are currently no treatments nor cures, the family launched a nonprofit to raise awareness and funds to work on L-CMD. For more information, visit www.lcmdresearch.org.

About Entos Pharmaceuticals Inc

A new reality in genetic medicine lies ahead, one that will be ushered in with the advent of safe, effective, and redosable nucleic acid delivery technologies. Since its inception in 2016, Entos^® has been dedicated to advancing next-generation genetic medicines using our proprietary Fusogenix™ PLV™ drug delivery system. The Fusogenix PLV platform is formulated with FAST proteins to enable the delivery of nucleic acid to target cells through direct fusion. Entos is pioneering the development of life-changing medicines for patients and has partnered with global companies, such as Eli Lilly, to accelerate and expand the impact of our platform. Entos Pharmaceuticals Inc. is headquartered in Edmonton, Canada, with its wholly owned U.S. and U.K. subsidiaries based in San Diego, California, and London, United Kingdom, respectively. For more information, visit www.entospharma.com, or follow Entos on LinkedIn. Entos^® word mark and design logo, Fusogenix^TM and PLV^TM are registered trademarks of Entos Pharmaceuticals Inc. All other trademarks and registered trademarks are the property of their respective owners.