Gene therapy developed at Duke to be commercialized by a local biotechnology company
Duke University recently signed an exclusive license with GeneVentiv Therapeutics, a Raleigh-based gene therapy company, to commercialize a gene therapy developed in the lab of Dwight Koeberl, Professor of Pediatrics in the Duke University School of Medicine.
The technology is the first universal gene editing therapy for infantile-onset and late-onset Pompe disease, a rare metabolic disease that causes the complex sugar glycogen to build up in cells. If untreated, this buildup is fatal.
With this partnership, GeneVentiv aims to bring this therapy – dubbed GENV-002 – into clinical trials and unlock the potential of treating patients with Pompe disease a decade earlier than current approaches.
“Infants have been left behind,” said Damon Race, CEO of GeneVentiv. “You haven’t been able to address them with the traditional AAV-based approaches and Dwight’s technology allows us to do that.”
“It’s really important that we’re able to partner with GeneVentiv because their expertise is complementary to ours,” said Koeberl. “We can move together really efficiently.”
“It’s bringing business and science together,” said Race.
The current standard of care is an enzyme replacement therapy also developed at Duke, by Professors Y. T. Chen, Priya Kishnani, and colleagues. It is sold under the brand names Myozyme and Lumizyme by Sanofi.
Duke’s research expertise in precision medicine for metabolic disorders also fits in the larger gene therapy strength of the Triangle. In fact, GeneVentiv previously licensed a universal gene therapy for hemophilia from UNC, which they have advanced to pivotal large animal studies.
“It’s great that a company can get access to technologies from both UNC and Duke,” said Robin Rasor, Associate VP for Translation & Commercialization, who negotiated the license through Duke’s Office for Translation & Commercialization. “We’re trying to build the ecosystem in the Triangle. There’s great science all over the area.”
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