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 it has received a Notice of Allowance from the U.S.
Patent and Trademark Office (USPTO) for U.S. Patent Application No.
18/161,560, titled “Engineered Meganucleases That Target Human
Mitochondrial Genomes.” Once issued, the patent arising from this
application will have a standard expiration date in April 2042.
The allowed composition of matter claims in this U.S.
application encompass a mitochondria-targeted ARCUS nuclease
(mitoARCUS) that is designed to specifically target, cleave, and
eliminate mutant mitochondrial DNA comprising an m.3243A>G
mutation. The m.3243A>G mutation is one of the most common
pathogenic mitochondrial DNA mutations, differing from wild-type
(normal) mitochondrial DNA by a single base change, and is
associated with the development of a number of disorders, including
primary mitochondrial myopathies that primarily affect skeletal
muscle, and mitochondrial encephalomyopathy, lactic acidosis and
stroke-like episodes (MELAS).
Precision recently announced PBGENE-PMM, the Company’s clinical
candidate targeting mutant mitochondrial DNA, as a potentially
first-in-class opportunity for treatment of m.3243 associated
primary mitochondrial myopathy. Utilizing the claimed mitoARCUS
nuclease, PBGENE-PMM is designed to target and eliminate mutant
mitochondrial DNA, allowing for repopulation by wild-type
mitochondrial DNA and restoration of mitochondrial function.
“The high specificity and single component nature of Precision’s
mitoARCUS nucleases are designed to enable specific elimination of
mutant mitochondrial DNA while allowing the normal mitochondrial
DNA to repopulate in the mitochondria and reestablish normal
function,” said Jeff Smith, PhD, Co-Founder and Chief Research
Officer at Precision BioSciences. “PBGENE-PMM holds the potential
to deliver a one-time, transformative treatment for patients with
primary mitochondrial myopathy.”
Unlike CRISPR-based gene editing tools, mitoARCUS nucleases are
able to gain access to mitochondria because they are small,
single-component proteins that integrate DNA-binding and
DNA-cleavage and do not require a nucleic acid, such as a guide
RNA, for targeting.
“The fact that mitoARCUS can be delivered directly to
mitochondria, and has the specificity to distinguish a single base
pair difference in the m.3243 A>G mutation, makes PBGENE-PMM a
very important potential treatment candidate for patients suffering
from m.3243 associated primary mitochondrial myopathy,” said Carlos
Moraes, PhD, Esther Lichtenstein Professor of Neurology, and Cell
Biology and Anatomy at the University of Miami Miller School of
Medicine, and co-inventor of the allowed application.
This U.S. application is jointly owned by Precision and the
University of Miami, which has exclusively licensed the rights to
the application to the Company.
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.
About Mitochondria and Primary Mitochondrial Myopathy
Mitochondria comprise multiple copies of a circular DNA referred
to as mitochondrial DNA, which encodes for 13 subunits of the
oxidative phosphorylation (OXPHOS) system, 2 rRNAs, and 22 tRNAs
that are all necessary to support mitochondrial function. It is
believed that a shift in mitochondrial DNA heteroplasmy toward
wild-type (normal) may provide therapeutic benefit for patients,
and not all mutant mitochondrial DNA must be eliminated to achieve
improvements in symptoms. Rather, mutant mitochondrial DNA levels
only need to be shifted below a disease threshold level.
Mitochondrial diseases that arise from mutations in
mitochondrial DNA are the most common hereditary metabolic
disorder, affecting 1 in 4,300 people. Primary mitochondrial
myopathy is characterized by severe fatigue and can affect skeletal
muscle, and other high energy organs such as the brain, eyes, ears
and heart. Primary mitochondrial myopathy currently lacks curative
treatment and impacts approximately 50% of patients with
mitochondrial disease.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. The Company intends such forward-looking statements to be
covered by the safe harbor provisions for forward-looking
statements contained in Section 27A of the Securities Act and
Section 21E of the Exchange Act. 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 expected safety, efficacy, and
benefit of our gene editing approaches including editing efficiency
and delivery methods, the suitability of ARCUS nucleases for gene
insertion, excision, and elimination, including the elimination of
mutant mitochondrial DNA, the clinical development, nomination, and
goals of our PBGENE-PMM program, the potential for a shift in
heteroplasmy toward wild-type mitochondrial DNA to restore
mitochondrial function, and therapeutic potential of an ARCUS gene
editing approach for the treatment of m.3243-associated
mitochondrial diseases. The words “aim,” “anticipate,” “approach,”
“believe,” “contemplate,” “could,” “estimate,” “expect,” “goal,”
“intend,” “look,” “may,” “mission,” “plan,” “possible,”
“potential,” “predict,” “project,” “promise,” “pursue,” “should,”
“target,” “will,” “would,” and other similar words or expressions,
or the negative of these words or similar words or expressions, are
intended to identify forward-looking statements, though not all
forward-looking statements use these words or expressions.
Forward-looking statements are based on management’s current
expectations, beliefs and assumptions and on information currently
available to us. These statements are neither promises nor
guarantees, but involve 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: our ability to become profitable; our ability to
procure sufficient funding and requirements under our current debt
instruments and effects of restrictions thereunder; risks
associated with raising additional capital; our operating expenses
and our ability to predict what those expenses will be; our limited
operating history; the success of our programs and product
candidates in which we expend our resources; our limited ability or
inability to assess the safety and efficacy of our product
candidates; our dependence on our ARCUS technology; the risk that
other genome-editing technologies may provide significant
advantages over our ARCUS technology; the initiation, cost, timing,
progress, achievement of milestones and results of research and
development activities, preclinical studies and clinical trials;
public perception about genome editing technology and its
applications; competition in the genome editing, biopharmaceutical,
and biotechnology fields; our or our collaborators’ ability to
identify, develop and commercialize product candidates; pending and
potential liability lawsuits and penalties against us or our
collaborators related to our technology and our product candidates;
the U.S. and foreign regulatory landscape applicable to our and our
collaborators’ development of product candidates; our ability to
obtain orphan drug designation or fast track designation for our
product candidates or to realize the expected benefits of these
designations; our or our collaborators’ ability to obtain and
maintain regulatory approval of our product candidates, and any
related restrictions, limitations and/or warnings in the label of
an approved product candidate; our or our collaborators’ ability to
advance product candidates into, and successfully design, implement
and complete, clinical trials; potential manufacturing problems
associated with the development or commercialization of any of our
product candidates; delays or difficulties in our and our
collaborators’ ability to enroll patients; changes in interim
“top-line” and initial data that we announce or publish; if our
product candidates do not work as intended or cause undesirable
side effects; risks associated with applicable healthcare, data
protection, privacy and security regulations and our compliance
therewith; the rate and degree of market acceptance of any of our
product candidates; the success of our existing collaboration
agreements, and our ability to enter into new collaboration
arrangements; our current and future relationships with and
reliance on third parties including suppliers and manufacturers;
our ability to obtain and maintain intellectual property protection
for our technology and any of our product candidates; potential
litigation relating to infringement or misappropriation of
intellectual property rights; our ability to effectively manage the
growth of our operations; our ability to attract, retain, and
motivate key executives and personnel; market and economic
conditions; effects of system failures and security breaches;
effects of natural and manmade disasters, public health emergencies
and other natural catastrophic events; effects of COVID-19 pandemic
and variants thereof, or any pandemic, epidemic or outbreak of an
infectious disease; effects of sustained inflation, supply chain
disruptions and major central bank policy actions; insurance
expenses and exposure to uninsured liabilities; effects of tax
rules; risks related to ownership of our common stock and other
important factors discussed under the caption “Risk Factors” in our
Quarterly Report on Form 10-Q for the quarterly period 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/20230927733715/en/
Mei Burris Senior Director of Finance and Corporate Controller
Mei.Burris@precisionbiosciences.com
Precision BioSciences (NASDAQ:DTIL)
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