- ARCUS showed durable, high-efficiency gene
insertion capabilities in NHPs and comparison to CRISPR-Cas9
- Data demonstrates ability of ARCUS to achieve
large gene excisions enabling significant functional muscle
improvement in a DMD mouse model
Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene
editing company utilizing its novel proprietary ARCUS® platform to
develop in vivo gene editing therapies for sophisticated gene
edits, including gene insertion, excision, and elimination, today
announced that the company will present two posters at the ESGCT
30th Annual Congress that highlight ARCUS’ differentiated ability
to make efficient, durable, and targeted insertion edits in
non-human primates (NHPs), as well as preclinical data
demonstrating the potential of ARCUS to achieve large excision of a
commonly mutated region of the dystrophin gene for the potential
treatment of Duchenne muscular dystrophy (DMD).
“We believe ARCUS has the potential to redefine gene editing by
enabling sophisticated gene edits such as gene insertion, excision,
and elimination,” said Jeff Smith, Chief Research Officer of
Precision BioSciences. “ARCUS’ ability to create 4 base pair, 3’
overhangs is designed to make predictable, highly consistent,
therapeutic edits and enable high efficiency gene insertion through
homology directed repair (HDR). In preclinical work presented today
at ESGCT, ARCUS demonstrated 17 times higher gene insertion
efficiency compared to CRISPR-Cas9, validating the importance of
the cut made by ARCUS. In addition, data presented at ESGCT
continue to support ongoing development of our gene excision
program focused on DMD, in development with Prevail Therapeutics, a
wholly-owned subsidiary of Eli Lilly and Company.”
Title: Unique features of ARCUS nucleases enable high
efficiency, targeted gene insertion in vivo Poster Number:
#P641 Presenter: Cassie Gorsuch, VP of Gene Therapy,
Precision Biosciences Date and Time: Wednesday, October 25,
2023, 5:00 PM - 6:15 PM CEST and Thursday, October 26, 2023, 8:30
PM - 9:30 PM CEST Location: Gare Maritime
In preclinical work presented today, ARCUS showed high
efficiency gene insertion in vitro (primary human T cells and
hepatocytes) and in vivo (infant and adult NHPs). ARCUS’ 3’
overhangs were shown to drive high efficiency gene insertion
compared to blunt cuts, and in vivo ARCUS demonstrated high
efficiency and durable insertion in newborn and infant NHPs when
used with OTC and F9 transgene insertion templates after
adeno-associated virus (AAV) delivery. ARCUS showed up to ~45%
insertion when administered by LNP along with an AAV carrying a
gene insertion template in adult NHPs, and demonstrated high
efficiency gene insertion via HDR in nondividing, primary human
hepatocytes.
Title: ARCUS-mediated excision of the “hot spot” region
of the human dystrophin gene results in functional improvement in a
mouse model of Duchenne muscular dystrophy (DMD) Poster
Number: Poster #P653 Presenter: Cassie Gorsuch, VP of
Gene Therapy, Precision Biosciences Date and Time:
Wednesday, October 25, 2023, 5:00 PM - 6:15 PM CEST and Thursday,
October 26, 2023, 8:30 PM - 9:30 PM CEST Location: Gare
Maritime
In the data on display today using early generation ARCUS
nucleases, Precision scientists observed the edited dystrophin
protein variant in multiple tissue types frequently involved in
progression of DMD, including heart, diaphragm, and skeletal
muscle. Furthermore, the maximum force output of the gastrocnemius
muscle in ARCUS-treated animals was significantly improved compared
to untreated mice, reaching 86% of the maximum force output levels
observed in non-diseased, control animals.
About Duchenne muscular dystrophy
DMD is a genetic disorder associated with mutations in the
dystrophin gene that prevent production of the dystrophin protein.
Dystrophin stabilizes the cell membrane during muscle contraction
to prevent damage, and the absence of intact dystrophin protein
leads to inflammation, fibrosis, and progressive loss of muscle
function and mass. Over time, children with DMD will develop
problems walking and breathing, eventually leading to death in the
second or third decade of life due to progressive cardiomyopathy
and respiratory insufficiency. DMD occurs in 1 in 3,500 to 5,000
male births, and currently there are limited approved therapies
available for patients.
About ARCUS
ARCUS is a proprietary genome editing technology discovered and
developed by scientists at Precision BioSciences. It uses
sequence-specific DNA-cutting enzymes, or nucleases, that are
designed to either insert (knock-in), excise (knock-out),
eliminate, or repair DNA of living cells and organisms. ARCUS is
based on a naturally occurring genome editing enzyme, I-CreI, that
evolved in the algae Chlamydomonas reinhardtii to make highly
specific cuts in cellular DNA and stimulate gene insertion at the
cut site by homologous recombination. Precision's platform and
products are protected by a comprehensive portfolio including
nearly 100 patents to date.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is an advanced gene editing company
dedicated to improving life (DTIL) with its novel and proprietary
ARCUS® genome editing platform that differs from other technologies
in the way it cuts, its smaller size, and its simpler structure.
Key capabilities and differentiating characteristics may enable
ARCUS nucleases to drive more intended, defined therapeutic
outcomes. Using ARCUS, the Company’s pipeline is comprised of in
vivo gene editing candidates designed to deliver lasting cures for
the broadest range of genetic and infectious diseases where no
adequate treatments exist. For more information about Precision
BioSciences, please visit www.precisionbiosciences.com.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements contained in this press release that do not
relate to matters of historical fact should be considered
forward-looking statements, including, without limitation,
statements regarding expected conference participation and
disclosure of preclinical data, the ability of ARCUS to make
predictable, highly consistent, therapeutic edits as well as
durable, high-efficiency gene insertion capabilities through HDR,
including NHPs, the potential to redefine gene editing by enabling
sophisticated gene edits such as gene insertion, excision, and
elimination, the clinical development, nomination, and goals of our
PBGENE-DMD program, therapeutic potential of an ARCUS gene editing
approach for the treatment of DMD, including ability of ARCUS to
achieve large gene excisions, enabling significant functional
muscle improvement in a DMD mouse model, and expected safety,
efficacy, and benefit of our gene editing approaches. In some
cases, you can identify forward-looking statements by terms such as
“aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,”
“estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,”
“plan,” “possible,” “potential,” “predict,” “project,” “pursue,”
“should,” “target,” “will,” “would,” or the negative thereof and
similar words and expressions.
Forward-looking statements are based on management’s current
expectations, beliefs and assumptions and on information currently
available to us. Such statements are subject to a number of known
and unknown risks, uncertainties and assumptions, and actual
results may differ materially from those expressed or implied in
the forward-looking statements due to various important factors,
including, but not limited to, the important factors discussed
under the caption “Risk Factors” in our Quarterly Report on Form
10-Q for the quarter ended June 30, 2023, as any such factors may
be updated from time to time in our other filings with the SEC,
which are accessible on the SEC’s website at www.sec.gov and the
Investors page of our website under SEC Filings at
investor.precisionbiosciences.com.
All forward-looking statements speak only as of the date of this
press release and, except as required by applicable law, we have no
obligation to update or revise any forward-looking statements
contained herein, whether as a result of any new information,
future events, changed circumstances or otherwise.
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version on businesswire.com: https://www.businesswire.com/news/home/20231026884837/en/
Investor and Media Contact: Mei Burris Senior Director of
Finance and Controller Mei.Burris@precisionbiosciences.com
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