When Damon Race, a Raleigh-based biopharmaceutical entrepreneur and investor, wanted to start a new company, he didn’t have to go far to find the founding technologies.
After looking at “30 to 40 assets” nationally, in October of 2020, Race chose to in-license a gene therapy devised within a half-hour’s drive at the University of North Carolina at Chapel Hill. In April 2025, he licensed a gene-editing therapy from Duke University, also a half-hour’s drive away, in Durham. 
Together, these gene therapy programs make up the product pipeline for GeneVentiv Therapeutics, a pre-clinical gene therapy company founded in 2020 in Raleigh.
There was no need to look further or to move elsewhere because the Research Triangle area has “a tremendous ecosystem for gene therapy innovation,” said Race, the company’s president and chief executive officer.
The UNC technology, developed by Pediatrics Professor Chengwen Li, M.D., Ph.D., uses adeno-associated viruses (AAVs) to ferry corrective genes into cells. GeneVentiv is developing it to treat hemophilia, a genetically inherited bleeding disorder.
The Duke technology, invented by Pediatrics Professor Dwight Koeberl, M.D., Ph.D., is a gene-editing platform that uses AAVs to deliver corrective genes into cells and a CRISPR Cas9 editor to precisely integrate those genes into cellular DNA. GeneVentiv is developing a combination platform to treat Pompe disease, an inherited metabolic disorder that causes developmental, muscular, and cardiovascular problems.
Both hemophilia A and Pompe are severe diseases without a one-and-done treatment option that gene therapy could provide.
“The therapies we have licensed share a common theme as universal therapies that are the first to address patients left behind by the shortcomings of other approaches,” Race said. “I wanted to develop a technology that was highly novel and impactful.”
If approved as safe and effective, the two gene therapies could potentially help about 270,000 people in the developed world at $3 million to $3.5 million per patient ─ a fraction of the cost of current therapies given over a lifetime, Race said.
Hemophilia therapy
Hemophilia is caused by a genetic deficiency of blood clotting proteins, called factors, resulting in internal or external bleeding, mostly in males. There are two types of the disease: type A, caused by low levels of factor VIII, and type B, caused by deficient levels of factor IX.
Inherited hemophilia A, the most prevalent form, occurs in about 1 of every 5,000 male births. An estimated 33,000 males in the United States live with the disorder.
The conventional way to treat hemophilia A and B is to give weekly injections of clotting factors or substitutes to compensate for the missing blood clotting factors so that the blood can clot properly.
More recently, gene therapies have been developed for hemophilia A and B. Both gene therapies deliver copies of the genes needed to restore normal clotting.
The FDA-approved hemophilia B gene therapy durably expresses the necessary clotting factor over time. However, the hemophilia A gene therapies are generally not durable, and patients can’t be re-dosed.
About 30% of hemophilia A patients and about 5% of hemophilia B patients develop neutralizing antibodies called inhibitors to the clotting factors. These patients are ineligible for gene therapies and “were being left behind by the gene therapies in development,” Race said.
GeneVentiv’s technology is intended to overcome these problems by introducing a different clotting factor, factor Va, that is durably expressed and restores blood clotting without triggering immune responses.
“We have the only durable gene therapy that is in development for hemophilia A with or without inhibitors,” Race said. “We are very proud of the fact that we are addressing patients who have been left behind.”
GeneVentiv is collaborating with UNC researchers to test the efficacy and safety of its one-time therapy in canine models. It collaborates with ReciBioPharm, a global contract development and manufacturing organization, to help with the complex manufacturing process ahead of a clinical trial.
After completing the pre-clinical studies, the therapy should enter human trials in late 2026 or early 2027, Race said.
A $2.5 million Small Business Research Innovation (SBIR) grant from the National Institutes of Health’s National Heart Lung Blood Institute is funding the therapy’s development in collaboration with UNC.
Pompe therapy
Pompe disease is caused by a deficiency of an enzyme, acid alpha-glucosidase, or GAA, that breaks down the complex sugar glycogen in cells. That leads to an excessive buildup of glycogen, causing muscle damage throughout the body.
“It’s a horrendous disease” that can be fatal, Race said.
About 25% of patients have infantile-onset Pompe, which manifests in the first few months of life, and 75% have late-onset Pompe, a less severe form that appears in childhood to adulthood. Both forms are rare diseases identified via newborn screening, and they affect about one in 40,000 people in the United States.
The standard treatment is enzyme replacement therapy, which requires lengthy intravenous infusions in a hospital every two weeks ─ a grueling regimen for infants and their parents.
GeneVentiv has the only gene-editing therapy in development to treat both forms of Pompe. The one-time therapy is designed to replace the faulty gene for the GAA enzyme with a functioning gene.
The therapy could enter clinical trials in late 2027 or early 2028, Race said. The company has applied for an SBIR grant to fund further development.
GeneVentiv has received letters of support for the therapy from Sarepta Therapeutics, Alexion Pharmaceuticals and AskBio, a pioneer in AAV gene therapy based in Research Triangle Park, N.C., and with roots at UNC.
Early funding critical
GeneVentiv operates virtually with a small staff of “less than 10,” Race said. It relies on sponsored university research and a network of advisors and consultants across the country.
The company’s business model is to identify and in-license gene therapy technologies that offer competitive advantages over other treatments. The company then de-risks and develops these therapies to advance them to approval by the U.S. Food and Drug Administration.
GeneVentiv’s early work was supported by a $50,000 KickStart commercialization grant from UNC and a $250,000 Small Business Research Loan from the North Carolina Biotechnology Center that funded proof-of-concept studies for the hemophilia gene therapy.
The grant and loan were “dominoes” in helping GeneVentiv close a seed round of financing, Race said.
“NCBiotech has an incredible due diligence process” that validates technologies transitioning out of academia and into startup companies, he said. “Without the work we were able to do with NCBiotech funding, we wouldn’t have had the data for a series seed investment, which carries us today.”
Jason Doherty, Ph.D., NCBiotech’s senior director of investments for emerging company development, said, “NCBiotech is proud to be an early supporter of GeneVentiv and its gene therapy approach to rare disease. Hemophilia and Pompe disease are devastating conditions with high unmet need and GeneVentiv brings a promising new approach that offers hope to patients and their families.”