November 2, 2020
RALEIGH, NC – GeneVentiv Therapeutics has entered a license agreement with University of North Carolina Chapel Hill for a gene therapy that is a universal cure for Hemophilia A or B patients with or without inhibitors to their missing clotting factors.
UNC has been prolific in spinning out successful gene therapy companies such as AskBio, StrideBio and Bamboo Therapeutics among others. As UNC’s latest spinout, GeneVentiv joins the ranks of promising North Carolina gene therapy companies.
Hemophilia is an inherited disorder that prevents production of factors necessary for normal blood clotting in response to injury. The current standard of care is weekly infusions of the missing clotting factors. One in three patients develop inhibiting anti-bodies that stop the treatments from working.
GeneVentiv has designed a gene to produce an activated form of Factor V. GeneVentiv’s therapy uses adeno-associated viruses to carry the therapeutic gene into human cells. AAVs are small viruses that can infect cells without causing any known diseases. The company has completed proof of concept studies to treat Hemophilia A and B with or without inhibitors and is preparing to conduct the remaining pre-clinical studies for an Investigational New Drug application.
“The hemophilia gene therapies currently in development can’t treat inhibitor patients. For inhibitor patients there has been no hope of a cure until now,” said Damon R. Race, who is GeneVentiv’s President and CEO.
To date, GeneVentiv has been funded by NIH grants and individuals. “There is tremendous inbound interest from leading gene therapy companies. As a result, we anticipate rapidly moving our hemophilia program forward, which is expected to enter the clinic mid-2022.” Said Race.