CureDuchenne’s commitment is a part of Gennao Bio’s Series A extension to help the company apply their gene monoclonal antibody (GMAB) platform technology to Duchenne muscular dystrophy (DMD) and other rare muscle diseases.
“We are honored to partner with Gennao Bio and leverage their innovative technology toward a potential cure for Duchenne and other rare muscle diseases. Through CureDuchenne Ventures, it is our mission to help accelerate scientific breakthroughs and this investment represents the next generation of potentially groundbreaking research to put an end to this devastating disease.”— Debra Miller, president of CureDuchenne Ventures and founder and chief executive officer of CureDuchenne
Delivers Nucleic Acid Therapeutics
Gennao’s GMAB platform technology can be used to efficiently deliver a broad range of nucleic acid payloads, including mRNA, DNA, siRNA and ASOs. The technology targets the nucleoside transporter ENT2, which is highly expressed on skeletal muscle cells thereby allowing for systemic administration and targeted delivery.
This non-viral delivery platform has the potential to overcome limitations of traditional gene delivery systems as it avoids issues with pre-existing antibodies and size limitations of viruses, allows for repeat dosing, and employs well-established manufacturing processes. Gennao Bio is developing this delivery system with an initial focus on addressing significant unmet needs in oncology, and now with this investment, a significant focus on rare muscle diseases, including DMD.
“We are honored to have a partnership and the support of CureDuchenne Ventures as we look to expand the research and development of our GMAB platform into rare skeletal muscle disease, including in DMD.”— Stephen Squinto, PhD, chief executive officer and chair of the board of Gennao Bio
[Source(s): CureDuchenne, PR Newswire]