Market Overview

Precision Biosciences Announces Collaboration with University of Pennsylvania to Develop Genome Editing Therapeutics


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

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
, 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

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