DURHAM, North Carolina, USA, September 04, 2018 – Precision BioSciences today announced an expansion of their ongoing collaboration with the Gene Therapy Program in the Perelman School of Medicine at the University of Pennsylvania (Penn) to pursue research and development of gene editing therapies. This relationship will allow Precision and Penn to design new in vivo gene therapies based on Precision’s proprietary ARCUS genome editing technology.
Regarding Precision’s work with Penn, CSO Derek Jantz stated, “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. 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 agreements between Precision and Penn include a Research, Collaboration and License Agreement (RCLA) with funding provided 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 knock-in or gene repair programs. The initial three-year program will include studies in non-human 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 already underway between Precision BioSciences and Penn have been published in the Nature Biotechnology article 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. 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.
About Precision BioSciences
Precision BioSciences is dedicated to improving life. Our mission is to cure genetic disease, overcome cancer, and feed the planet. We are striving to achieve this goal with ARCUS, our therapeutic-grade, naturally-derived genome editing system that combines both specificity and efficacy to help overcome life’s greatest genetic challenges. For additional information, please visit www.precisionbiosciences.com.
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