CureDuchenne Commits $1M to Gennao Bio for Rare Muscle Disease Research

Investment to Expand First-in-Class, Nucleic Acid Therapeutics Towards Targeting Muscle Diseases

Newport Beach, Calif. (December 22, 2021) – CureDuchenne Ventures, a subsidiary of CureDuchenne, a leading global nonprofit focused on finding and funding a cure for Duchenne muscular dystrophy, announces a $1 million investment in Gennao Bio. 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,” said Debra Miller, president of CureDuchenne Ventures and founder and chief executive officer of CureDuchenne. “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.”

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,” said Stephen Squinto, Ph.D., chief executive officer and chair of the board of Gennao Bio.

CureDuchenne Ventures is the investment arm of CureDuchenne, which deploys donor dollars to fund Duchenne muscular dystrophy treatments with the goal of funding cures for the entire Duchenne community. CureDuchenne’s robust pipeline of therapies comes from a diverse array of cutting-edge technologies used to treat all aspects of Duchenne. A successful investment made by CureDuchenne Ventures is a success for the entire Duchenne community, with 90 percent of earnings re-invested back into Duchenne research to further the goal of a cure. For more information on CureDuchenne Ventures, please visit

About CureDuchenne
CureDuchenne is recognized as a global leader in research, patient care and innovation for improving and extending the lives of those with Duchenne muscular dystrophy.  As the leading genetic killer of young boys, Duchenne affects more than 300,000 individuals living today. CureDuchenne is dedicated to finding and funding a cure for Duchenne by breaking the traditional charitable mold through an innovative venture philanthropy model that funds groundbreaking research, early diagnosis, and community education. For more information on how to help raise awareness and funds needed for research, please visit  

About Gennao Bio
Gennao Bio is a privately held genetic medicines company developing first-in-class targeted nucleic acid therapeutics utilizing a proprietary gene monoclonal antibody (GMAB) platform technology. GMAB is an adaptive technology that uses a cell-penetrating antibody to non-covalently bind to and deliver therapeutic levels of a wide variety of nucleic acid payloads to select cells. This non-viral delivery platform is differentiated from traditional gene delivery systems as it can deliver multiple types of nucleic acids, allows for repeat dosing and employs well-established manufacturing processes. Gennao Bio is developing this delivery system with an initial focus in oncology and rare monogenic skeletal muscle diseases.

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