Newswise — PHILADELPHIA—(Sept. 3, 2015)—The Wistar Institute, an international leader in biomedical research in cancer, immunology and vaccines, and Spark Therapeutics, a gene therapy leader seeking to develop one-time, life-altering treatments for debilitating genetic diseases, announced today a collaboration designed to improve the durability of gene therapy treatments targeting the liver by advancing new ways to manage the immune response to treatment. The research collaboration combines Wistar’s strong expertise in viral immunology with Spark’s industry-leading gene therapy research and development capabilities.
The field of gene therapy centers on delivering normal genes into patients to target or replace defective genes in inherited and rare diseases as well as cancers. World-renowned hematologist Katherine A. High, M.D., Spark Therapeutics’ co-founder, president and chief scientific officer, and preeminent immunologist Hildegund C.J. Ertl, M.D., professor and director of The Wistar Institute Vaccine Center, will work together under a two-year collaboration to advance gene therapy technologies for liver-targeted indications, such as hemophilia.
This newly formed collaboration is the evolution of 15 years of joint research between Drs. High and Ertl, and several academic institutions in the Philadelphia region and beyond.
“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,” said Dr. High. “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 adeno-associated virus (AAV)-based therapeutics in liver-directed gene therapy. We at Spark are proud to collaborate with Dr. Ertl, an acknowledged leader in viral immunology and immune responses to vectors.”
Prior collaboration between Drs High and Ertl has centered on elucidating and better managing cell-mediated human immune response to gene therapy vectors. This response can function as a barrier to long-term expression in gene therapy if it is not appropriately managed and controlled.
“Evaluating immune responses to a new viral vector is challenging,” said Dr. Ertl. “The immune system evolved to get rid of pathogens and, unfortunately, takes the same approach to virus-based gene transfer vehicles. Nevertheless, the biology of a viral vector, especially one based on an AAV, is fundamentally different from that of a rapidly spreading virus. Accordingly, the ensuing immune responses are distinct and it is quite fascinating to figure out these differences. 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.”
“Meaningful industry-academia collaborations are critical for advancing innovative therapies,” said Dario C. Altieri, M.D., president and CEO of The Wistar Institute, director of Wistar’s Cancer Center, and the Robert and Penny Fox Distinguished Professor. “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.”
The Wistar Institute is an international leader in biomedical research with special expertise in cancer research and vaccine development. Founded in 1892 as the first independent nonprofit biomedical research institute in the country, Wistar has held the prestigious Cancer Center designation from the National Cancer Institute since 1972. The Institute works proactively to ensure that research advances move from the laboratory to the clinic as quickly as possible. Wistar’s Business Development team is dedicated to advancing Wistar Science and Technology Development through creative partnerships. www.wistar.org.
Spark Therapeutics is a gene therapy leader seeking to transform the lives of patients suffering from debilitating genetic diseases by developing one-time, life-altering treatments. Spark’s integrated gene therapy platform builds on two decades of research, development and manufacturing at The Children’s Hospital of Philadelphia, including human trials conducted across diverse therapeutic areas and routes of administration. www.sparktx.com
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