CureDuchenne Invests $1M in Gennao Bio’s Effort to Find Therapies

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by Mary Chapman |

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Gennao Bio | Muscular Dystrophy News | CureDuchenne Ventures | Illustration of a piggy bank

Genetic medicines company Gennao Bio will use a $1 million CureDuchenne Ventures investment to help expand its gene monoclonal antibody (GMAB) platform technology, which is now applied to rare muscle disorders, including Duchenne muscular dystrophy (DMD).

The initial focus of the nonviral delivery system Gennao Bio is developing had been significant unmet needs in oncology. CureDuchenne’s commitment, which is part of Gennao Bio’s Series A funding extension, will allow it to extend that focus to DMD and related diseases.

Using a proprietary platform that differs from traditional gene delivery systems, Gennao Bio seeks to develop first-in-class therapeutics through an adaptive technology that uses a cell-penetrating antibody to bind to and deliver to certain cells therapeutic amounts of a broad range of nucleic acid payloads. Nucleic acids are made out of units called nucleotides and include DNA and RNA.

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“We are honored to partner with Gennao Bio and leverage their innovative technology toward a potential cure for Duchenne and other rare muscle diseases,” Debra Miller, president of CureDuchenne Ventures and founder and CEO of CureDuchenne, said in a press release. “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.”

Through efficient delivery of an array of nucleic acid payloads, including messenger RNA (mRNA), DNA, small interfering RNA (siRNA), and antisense oligonucleotides (ASOs), Gennao’s GMAB platform targets the nucleoside transporter ENT2. Because the transport protein is highly expressed on tumor cells and skeletal muscle, it permits systemic administration and targeted delivery.

mRNA is an intermediate molecule in protein production from genes, siRNA is a synthesized RNA molecule used to temporarily block the activity of a given gene, and ASOs are also synthesized molecules used to specifically bind to a target RNA.

“Our gene monoclonal antibody technology (GMAB) has several unique attributes that enable it to systematically deliver multiple types of nucleic acids, directly into the cell, while preserving therapeutic function,” Gennao Bio states on its website.

By not using viruses as delivery vehicles of therapies, the platform skirts issues with pre-existing antibodies and viral size, permits repeat dosing, and uses established manufacturing processes to surmount constraints posed by conventional gene delivery systems, according to the company.

“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, PhD, Gennao Bio’s CEO and board chairman.

A global leader in research, patient care, and innovation, CureDuchenne works to find and fund a cure for Duchenne muscular dystrophy. CureDuchenne Ventures uses donations to fund DMD therapies with the aim of finding cures.