Fortress Biotech subsidiary Aevitas Therapeutics enters into gene therapy research project with University of Pennsylvania

Fortress Biotech (NASDAQ:FBIO) today revealed that its subsidiary Aevitas Therapeutics Inc has entered a ground-breaking research agreement involving its gene therapy with the University of Pennsylvania’s Perelman School of Medicine.

The tie-up, which will be led by the research lab of Dr Wenchao Song, a pharmacology professor, is aimed at evaluating Aevitas’ adeno-associated virus gene therapy technology in Song’s animal models of so-called complement-mediated diseases.

The research will investigate dosing and attempt to confirm that this gene therapy is restoring the lasting production of proteins that regulate the alternative pathway in the complement system, a multimolecular system composed of proteins and cell membrane receptors that supports the way the immune system functions.

Irregularities in these proteins may play a role in several complement-mediated diseases such as age-related macular degeneration.

“Further validation of Aevitas’s AAV gene therapy in animal models of complement-mediated diseases is a key step in advancing meaningful therapies toward the clinic,” noted Dr. Lindsay Rosenwald, Fortress Biotech’s  CEO, in a statement. “We look forward to working with Dr Song, a world-renowned leader in the pathogenesis of complement-mediated diseases, to advance gene therapies with the potential to provide long-term benefit to patients in need.”

The New York-based copmpany’s shares were trading down 5.3% at US$1.79 in early afternoon trade.

Aevitas Therapeutics aims to use its particular type of gene therapies to develop cures for so-called complement-mediated diseases such as atypical hemolytic uremic syndrome, a disease that causes abnormal blood clots to form in small blood vessels in the kidneys, as well as macular degeneration and paroxysmal nocturnal hemoglobinuria, a life-threatening blood disease.

Story by ProactiveInvestors