A new partnership aims to change the game in gene therapy for Duchenne muscular dystrophy (DMD), as CureDuchenne Ventures invests $1 million in Canadian biotech Entos Pharmaceuticals to develop a redosable therapy capable of delivering full-length dystrophin.

The funding will support the use of Entos’ non-viral Fusogenix PLV platform to engineer a muscle-targeted therapy for DMD, a rare and severe genetic disorder that causes progressive muscle degeneration in boys. The platform, which uses proteolipid vehicles (PLVs), is designed to deliver genetic material, including RNA, DNA, and gene editing payloads, without relying on adeno-associated viruses (AAVs). Current AAV-based approaches are known to carry redosing limitations and immune response risks.

“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 organization said its funding strategy is driven by donations from the patient community and aims to de-risk novel approaches with the potential to bring safe and effective treatments to patients faster.

Entos’ CEO, John Lewis, said: “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. We’re hopeful this approach will make a real difference for people with Duchenne muscular dystrophy and their families.”

The ability to deliver full-length dystrophin has long been considered a critical goal in the treatment of DMD. Most current gene therapies deliver only shortened versions of the protein due to size constraints associated with viral vectors. Entos believes its PLV system could overcome these technical limitations while avoiding the risks associated with immune response and one-time-only dosing.