Our recent investment in Entos Pharmaceuticals (https://entospharma.com) aims to explore the potential of their non-viral, redosable Fusogenix PLV platform to deliver full-length dystrophin protein to all muscle groups in a safe, and effective manner with lower cost compared to an adeno-associated virus (AAV) (https://entospharma.com/news-%26-media/f/entos-to-develop-dystrophin-therapy-for-dmd-with-cureduchenne).
Gene therapy is a significant therapeutic option for individuals with DMD. While AAV viral vectors have facilitated initial advancements, they present limitations such as restricted carrying capacity necessitating the delivery of a micro-dystrophin protein, safety concerns related to the high dose of AAV required, and the inability to redose due to neutralizing antibodies. These challenges underscore the need for alternative gene delivery methods to provide safer and more effective therapies.
Non-viral systems, with their safer profiles, scalability, capability to deliver larger gene transcripts and the potential for redosing, represent a promising avenue for future developments.
Entos’ Fusogenix PLV platform is a delivery system that combines aspects of both viral and non-viral approaches. It employs a new mechanism for intracellular delivery of RNA, DNA, and gene editing therapies. Our funding will be used to develop a redosable, muscle-targeted Fusogenix PLV therapeutic to deliver full-length dystrophin for the treatment of DMD.
See press release HERE
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