CureDuchenne Ventures Invests $1 Million in Entos

Rare Daily Staff

CureDuchenne Ventures, the venture philanthropy arm of CureDuchenne, said it made an initial $1 million investment in Entos Pharmaceuticals to create a muscle-targeting therapeutic to deliver full-length dystrophin for the treatment of the rare neuromuscular disease Duchenne muscular dystrophy.

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. As muscle weakness and degeneration progress to their upper limbs, they will lose the ability to perform other activities of daily living.  Cardiopulmonary complications are the primary cause of death.

Entos’ Fusogenix PLV platform is a delivery system that combines the best aspects of viral and non-viral approaches. It uses a new mechanism for intracellular delivery for a range of genetic medicines, including RNA, DNA, and gene editing therapies.

“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.”

Though adeno-associated viruses are widely used as vectors for gene therapies, they are not redosable because patients can develop antibodies to them once exposed. They are also too small to carry the full dystrophin gene. As a result, gene therapy developers have used microdystrophin, a truncated form of dystrophin gene.

Debra Miller, founder and CEO of CureDuchenne, said Entos’ technology has the potential to overcome the limitations of current AAV-based approaches and treat people with pre-existing immunity, allow for redosing, and deliver full-length dystrophin to all of the necessary muscle tissues.

“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,” she said.