GeneVentiv Announces Global Licensing Deal for Pompe Disease Gene Editing Platform
RALEIGH, N.C., /PRNewswire/ — GeneVentiv Therapeutics today announced a global licensing agreement with Duke University for GENV-002, a groundbreaking gene editing therapy designed to treat both infantile-onset and late-onset Pompe disease, affecting an estimated 120,000 people in the developed world.
Pompe disease, caused by a deficiency in the acid-alpha-glucosidase (GAA) enzyme, leads to severe muscle weakness, respiratory failure, and cardiac complications due to glycogen buildup. Current Enzyme Replacement Therapy (ERT) is burdensome, requiring bi-weekly infusions and costing millions over a lifetime, with no curative option.
GENV-002, developed by Dr. Dwight Koeberl of Duke University, utilizes an adeno-associated virus (AAV) delivery system combined with CRISPR-Cas9 gene editing to integrate a healthy donor gene into liver cells. This liver depot strategy overcomes limitations of traditional AAV therapies, enabling treatment of all Pompe disease patients, including infants.
“GENV-002 represents a significant advancement, potentially allowing for early intervention in all Pompe disease cases,” said Damon Race, CEO of GeneVentiv Therapeutics. “This therapy, combined with newborn screening, can be administered in infancy, years ahead of current approaches.” “GENV-002 surpasses existing AAV gene therapies by offering a universal treatment for both infantile and late-onset Pompe disease through gene editing,” added Paris Margaritis, CEO of GeneVentiv Therapeutics. “Similar to our lead program, GENV-HEM, which addresses all hemophilia types, GENV-002 aims to provide a transformative solution for patients.”
The technology was licensed through the Duke University Office for Translation & Commercialization. GeneVentiv aims to bring this potentially curative therapy to the market, addressing a significant unmet medical need.
https://www.prnewswire.com/news-releases/geneventiv-announces-global-licensing-agreement-for-universal-gene-editing-therapy-for-infantile-onset-and-late-onset-pompe-disease-302422876.html
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