Precision BioSciences, UPenn to Collaborate on Genome Editing Therapeutics Development

September 4, 2018
Precision BioSciences, UPenn to Collaborate on Genome Editing Therapeutics Development
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Precision BioSciences announced an expansion of its  collaboration with the Gene Therapy Program in the Perelman School of Medicine at the University of Pennsylvania (UPenn) for the research and development of gene editing therapies. The collaboration will leverage the ARCUS gene editing technology of Precision BioSciences to allow it and UPenn to design new in vivo gene therapies 

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 Derek Jantz, CSO of Precision BioSciences in a press release. “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.”

The three-year collaboration will encompass three gene knock-out programs and up to another three programs focused on knock-in or gene repair.

Research efforts of the two organizations have already yielded a paper Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol, which details efforts to develop an in vivo liver editing vector to disable the cholesterol-regulating gene PCSK9, published July 8 in Nature Biotechnology. 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 in rhesus macaques.

“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, M.D. Ph.D., 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.”