This article is more than 5 years old. Information may no longer be current.
Wistar Institute collaborates with Spark Therapeutics for liver-directed gene therapy
Click Here to Manage Email Alerts
The Wistar Institute announced it has entered into a research collaboration with Spark Therapeutics Inc. to improve gene therapy treatments that target the liver through creating and managing immune responses to treatment, according to a press release.
Katherine A. High, MD, co-founder, president and chief scientific officer of Spark Therapeutics, will work with Hildegund C.J. Ertl, MD, director of The Wistar Institute Vaccine Center, under a 2-year collaboration to advance gene therapy technologies, specifically those associated with adeno-associated virus (AAV) vectors, for liver-targeted indications, such as hemophilia, according to the release.
“I am extremely pleased that we will be able to continue these studies with our Wistar colleagues, following many years of collaborating at The Children’s Hospital of Philadelphia,” High said in the release. “A better understanding of the mechanisms underlying the human immune response to AAV vectors and the development of more effective strategies to manage this response will create important opportunities to widen the application of AAV-based therapeutics in liver-directed gene therapy.”
Ertl stated in the release: “Our goal is to define immune responses to AAV vectors, identify their Achilles’ heel, and then use this knowledge to devise strategies that put a lid on them. This, in turn, may allow for sustained expression of the therapeutic protein without having to immunosuppress our gene transfer recipients.”
High and Ertl have collaborated in the past and focused on finding better ways to manage the human immune response to gene therapy vectors, which can serve as a barrier for long-term expression in gene therapy, according to the release.
“We are pleased to be working with Spark and are hopeful that our collective contributions will have an impact on the future of gene therapy,” Dario C. Altieri, MD, president and CEO of The Wistar Institute, said in the release.
Disclosures: High reports being employed by Spark Therapeutics; and Ertl and Altieri report being employed by The Wistar Institute.