GenVec, Inc., has entered into an exclusive “option agreement” with Washington University (WU) in St. Louis to license intellectual property and technology related to specific research by WU investigators in gene editing and pulmonary endothelial cell targeting.
GenVec is a Maryland-based biopharmaceutical company that specializes in genetically based therapies and vaccines. The license would allow the company to use technology developed by researchers David T. Curiel, MD, PhD, WU professor of radiation oncology, and Jeffrey Arbeit, MD, professor of surgery at the WU School of Medicine. GenVec plans to initially focus on the development of hemophilia treatments using its AdenoVerse™ platform, combined with additional technologies developed at WU.
AdenoVerse™ includes a catalog of GenVec’s proprietary adeno-associated viral vectors (AAVs), which are the vehicles that carry the genetic instructions to illicit the production of therapeutic proteins such as factor VIII (FVIII) and factor IX (FIX). AAVs have been shown to successfully deliver the genetic material into living cells to desired therapeutic effect without causing disease or triggering significant immune responses.
In addition, findings of the WU researchers suggest that endothelial cells that line blood vessels in the lungs can be used as production sources of FVIII or FIX. Genetic editing, another novel technique, allows investigators to further target a specific location within these cells to generate sustained production of therapeutic proteins.
“This agreement provides GenVec with the foundation to establish a proprietary and differentiated program using an individual’s pulmonary endothelium as a site for protein production,” said Douglas Swirsky, president and CEO of GenVec. “Proprietary vectors from our AdenoVerse platform are well suited for the delivery of gene editing payloads and could be useful in emerging therapeutic approaches to the long-term correction of genetic disorders such as those that cause blood factor deficiencies.”
“Combining gene editing and pulmonary endothelial cell targeting approaches with GenVec’s AdenoVerse technology offers a unique and compelling platform to provide patients with proteins that they are deficient in to potentially address hemophilia and numerous other unmet medical needs,” added Douglas E. Brough, PhD, chief scientific officer of GenVec.
Source: PRNewswire, January 5, 2017
