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CureDuchenne Ventures Invests in Entos Pharmaceuticals for Development of a Redosable, Full-Length Dystrophin Gene Therapy


News provided by

CureDuchenne

May 22, 2025, 08:00 ET

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(PRNewsfoto/CureDuchenne)
(PRNewsfoto/CureDuchenne)
Entos Pharmaceuticals
Entos Pharmaceuticals

NEWPORT BEACH, Calif. and EDMONTON, AB , May 22, 2025 /PRNewswire/ -- CureDuchenne Ventures announced an initial $1M investment in Entos Pharmaceuticals Inc (Entos), a biotech company that develops genetic medicines utilizing its non-viral, redosable Fusogenix PLV delivery platform. Entos will use this funding to create a muscle-targeting therapeutic to deliver full-length dystrophin for the treatment of Duchenne muscular dystrophy (DMD). 

Entos' Fusogenix PLV platform is a delivery system that combines the best aspects of viral and non-viral approaches. Using a completely new mechanism for intracellular delivery of RNA, DNA and gene editing therapies, Fusogenix PLV is suitable for a wide range of best-in-class genetic medicines. Entos is committed to using this technology to develop cures and improve the lives of patients and their families. 

Entos will share information about this platform and their plans in Duchenne with families at the CureDuchenne 2025 FUTURES National Conference, held on May 22-25 in San Antonio, Texas. The session will be held on Friday, May 23 at 11:50 a.m. CT and will also be livestreamed.

"We are very excited by the potential of Entos Pharmaceuticals' technology and look forward to collaborating with Entos in pursuit of a gene therapy that overcomes the limitations of current AAV-based approaches—one that would not exclude individuals with pre-existing immunity, allow for redosing, and, crucially, deliver full-length dystrophin to all of the necessary muscle tissues," said Debra Miller, founder and CEO of CureDuchenne. "CureDuchenne has long been a leader in funding innovative therapeutic approaches, and this investment underscores our continued use of venture philanthropy to catalyze progress towards transformative treatments for Duchenne. We extend our heartfelt gratitude to our donors and the Duchenne community—your support has made this investment possible and continues to drive progress toward a cure."

"The Fusogenix PLV platform enables the safe, effective, and redosable delivery of full-length functional genes, like dystrophin, to muscle cells throughout the body. Given that Fusogenix PLV-based treatments can be given more than once, if needed, this could be a real game-changer," said John Lewis, CEO of Entos Pharmaceuticals. "We're hopeful this approach will make a real difference for people with Duchenne muscular dystrophy and their families."

About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is a rare, progressive, inherited neuromuscular disease that occurs in approximately one in every 3,500-5,000 males. It is caused by a mutation in the gene that encodes instructions for dystrophin, a key structural protein in muscle. Symptoms of DMD usually appear in infants and toddlers as difficulty in walking, climbing stairs, or standing from a sitting position. Most individuals with DMD will require full-time use of a wheelchair in their teens, and as muscle weakness and degeneration progresses to their upper limbs, lose the ability to perform other activities of daily living.  Cardiopulmonary complications are the primary cause of death.

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™ and PLV™ are registered trademarks of Entos Pharmaceuticals Inc. All other trademarks and registered trademarks are the property of their respective owners.

About CureDuchenne Ventures  CureDuchenne Ventures, LLC is the investment arm of CureDuchenne, a non-profit patient advocacy organization and global leader in funding research, patient care, and innovations for improving and extending the lives of those with Duchenne muscular dystrophy (DMD). CureDuchenne Ventures has supported transformative treatments for DMD, including 19 projects that advanced to human clinical trials, and multiple equity investments in companies striving to overcome the limitations of first-generation exon-skipping and gene therapies. For more information, please visit cureduchenne.org. 

SOURCE CureDuchenne

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