- Data presented at Mitochondrial Medicine
Therapeutic Development (MMTD) annual conference demonstrated
ARCUS’ ability to efficiently eliminate mutant mitochondrial DNA
without nuclear off-target editing
- PBGENE-PMM shifted heteroplasmy by
eliminating mutant m.3243 mitochondrial DNA (mtDNA) while
repopulating wild type mtDNA, leading to improved mitochondrial
function in cells
- Precision expects to submit a CTA and/or IND
for PBGENE-PMM in 2025
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 elimination, insertion, and excision, today
announced a poster presentation highlighting new data for the
Company’s PBGENE-PMM program, evaluating an ARCUS nuclease as a
potential treatment for m.3243-associated primary mitochondrial
myopathy (PMM). These data are being presented at the Mitochondrial
Medicine – Therapeutic Development Annual Conference, being held
from March 18-20, 2024 in Hinxton, UK.
“Today’s data from our PBGENE-PMM program further validate our
work with ARCUS and continue to demonstrate its ability to make
precise edits while avoiding off-target activity,” said Jeff Smith,
PhD, Chief Research Officer of Precision BioSciences. “We believe
ARCUS is uniquely suited for editing mitochondrial DNA due to its
ability to discriminate a single nucleotide difference, making it
ideal for editing point mutations such as m.3243. PBGENE-PMM is
designed to eliminate mutant mitochondrial DNA leaving normal
functioning wild-type mitochondrial DNA intact to repopulate the
cell, resulting in a shift in heteroplasmy and improvement in
mitochondrial function.”
Smith continued, “Additionally, ARCUS can cross mitochondrial
membranes in order to access the mitochondrial DNA. This is
possible because ARCUS is a protein-only editor with both
recognition and catalytic activity all in one single protein that
does not require a guide-RNA. This is not possible for
CRISPR-derived editors such as CRISPR-Cas, Base and Prime editors.
We look forward to advancing PBGENE-PMM towards clinical readiness
this year and anticipate filing a CTA and/or IND submission in
2025.”
Details for the presentation are as follows:
Title: Shifting m.3243A>G heteroplasmy with
PBGENE-PMM: Gene editing therapy for primary mitochondrial myopathy
Poster: P22 Presenter: Wendy Shoop, PhD, Research
Lead, Precision Biosciences Date and Time: Tuesday, March
19, 2024, 6:00-7:00 PM GMT Location: Hinxton Hall Conference
Centre, Wellcome Genome Campus, U.K.
In preclinical work presented today, ARCUS demonstrated highly
selective elimination of mutant m.3423G mtDNA. PBGENE-PMM, which
contains both a mitochondrial targeting sequence and a nuclear
export signal, was found to localize exclusively to mitochondria
and without any detectable off-target editing in the nuclear
genome. As the m.3243A>G mutation only differs from the
wild-type sequence by a single nucleotide, PBGENE-PMM was optimized
to prevent cutting of wild-type mtDNA while maintaining activity
against mutant mtDNA. When evaluated in cells that contain
heteroplasmic m.3243A>G mtDNA, PBGENE-PMM-treated cells were
found to contain 0.3% mutant mtDNA three days post-transfection,
compared to control cells which contained 95% mutant mtDNA. This
robust shift in heteroplasmy resulted in a nearly two-fold increase
in both basal and maximal respiration. Together, these data support
the development of PBGENE-PMM as a single-treatment, in vivo gene
editing therapeutic for m.3243-associated primary mitochondrial
myopathy.
About PBGENE-PMM
PBGENE-PMM is our wholly-owned, first of its kind treatment for
m.3243 associated primary mitochondrial myopathy (PMM).
Mitochondrial diseases are the most common hereditary metabolic
disorder, affecting 1 in 4,300 people. PMM currently lacks a
curative treatment and impacts approximately 50% of patients with
mitochondrial disease. In the Company’s 2023 publication in Nature
Metabolism, Precision presented new data highlighting the high
specificity and single component nature of the PBGENE-PMM and
ability to specifically edit and eliminate mutant mitochondrial DNA
while allowing wild-type (normal) mitochondrial DNA to repopulate
in the mitochondria, thus restoring normal function. Precision
expects to submit a CTA and/or IND for this program in 2025.
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 more
than 130 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.
ARCUS is a highly precise and versatile genome editing platform
that was designed with therapeutic safety, delivery, and control in
mind. 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 the therapeutic potential of an ARCUS gene
editing approach for the treatment of m.3243-associated PMM,
including the ability of ARCUS to preferentially target and
eliminate mutant m.3243G mtDNA with high specificity and without
off-target activity, anticipated timing of a CTA and/or IND filing,
the ability of mitoARCUS to shift heteroplasmy, and expected
safety, efficacy, and benefit of our gene editing approaches.. In
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Forward-looking statements are based on management’s current
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and success of our programs and product candidates in which we
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other genome-editing technologies may provide significant
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disease; the success of our existing collaboration agreements, and
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effects of natural and manmade disasters, public health emergencies
and other natural catastrophic events; effects of sustained
inflation, supply chain disruptions and major central bank policy
actions; market and economic conditions; risks related to ownership
of our common stock, including fluctuations in our stock price; our
ability to meet the requirements of and maintain listing of our
common stock on Nasdaq or other public stock exchanges; and other
important factors discussed under the caption “Risk Factors” in our
Quarterly Report on Form 10-Q for the quarterly period ended
September 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 https://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/20240319209835/en/
Investor and Media Contact: Naresh Tanna Vice President,
Investor Relations Naresh.Tanna@precisionbiosciences.com
Precision BioSciences (NASDAQ:DTIL)
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