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U Penn expands gene therapy partnership with Precision Biosciences


By David Schwartz
Published: September 11th, 2018

The University of Pennsylvania’s Perelman School of Medicine and Precision BioSciences announced an expansion of their ongoing gene therapy collaboration, which is focused on designing new in vivo gene therapies based on Precision’s proprietary ARCUS genome editing technology.

The deal includes a Research, Collaboration and License Agreement (RCLA) that provides funding to Penn and a license to certain technology invented under the RCLA. The collaboration will include three gene knock-out programs and up to three gene repair programs. The initial research will include studies in primate models with the goal of bringing candidates to IND during that time.

“The combination of Precision’s genome editing technology and our experiences in gene delivery and translational research provides a unique opportunity to advance our understanding of innovative in vivo genome editing approaches,” said James M. Wilson, MD, PhD, professor of medicine and pediatrics at the Perelman School of Medicine. “The platform technology developed by Precision to create such active and specific editing nucleases is impressive.”

Studies conducted by Precision BioSciences and Penn have already shown effectiveness in rhesus macaques to disable the cholesterol-regulating gene PCSK9. The authors reported that a single administration of an AAV vector encoding a PCSK9-directed ARCUS nuclease resulted in long-term reductions in serum PCSK9 and LDL cholesterol.

“We are excited to expand our collaboration with Jim Wilson and the Penn Gene Therapy Program. The Wilson laboratory is unparalleled in its ability to translate cutting-edge gene therapy technology into potential treatments for patients in need,” said Precision Chief Science Officer Derek Jantz. “The team’s proven success identifying gene therapy candidates and advancing them through preclinical development allows them to provide the critical insight needed to help move ARCUS into the clinic.”

Source: SC Now

Posted under: Industry-Sponsored Research Week

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