- Company highlights new preclinical data for
wholly-owned and partnered programs following prioritization of in
vivo gene editing portfolio
- Expands portfolio with PBGENE-PMM as a
potentially curative treatment for primary mitochondrial
myopathy
- Virtual R&D Day featuring key opinion
leaders and members of Precision’s leadership and scientific teams
to be held today at 9:00 AM ET
Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene
editing company utilizing its novel proprietary ARCUS® platform to
develop in vivo gene editing therapies, today announced that the
Company plans to highlight its current development programs,
pipeline updates, and underlying ARCUS platform technology during a
virtual R&D Day to be held today at 9:00 AM ET.
“At Precision, we are moving forward with a renewed identity and
focus on our core foundational strength as a gene editing company.
During today’s R&D Day, we are excited to highlight the
potential of our ARCUS genome editing platform to develop
differentiated programs enabled by the nature of ARCUS’ unique cut,
size, and simplicity,” said Michael Amoroso, Chief Executive
Officer at Precision BioSciences. “In addition to illustrating
where we see a clear advantage for ARCUS, we will drill down on the
novel attributes of ARCUS that inform our development program
strategy. We look forward to highlighting the progress we are
making across our robust wholly-owned and partnered in vivo gene
editing portfolio.”
“As we continue advancing our lead program PBGENE-HBV targeting
hepatitis B virus (HBV) toward a 2024 clinical trial application
(CTA) and/or investigational new drug (IND) application, we are
pleased to introduce PBGENE-PMM, our first development candidate
targeting mitochondrial DNA. PBGENE-PMM holds the potential to
deliver a one-time, transformative treatment for patients with
primary mitochondrial myopathy (PMM),” said Jeff Smith, Co-Founder
and Chief Research Officer. “Together with PBGENE-HBV, we believe
that both of these programs are emblematic of the unique advantages
of our ARCUS technology. As we look ahead, we plan to drive organic
development of programs focusing on more sophisticated edits such
as viral or mutant mitochondrial genome elimination, defective gene
excision, and gene insertion with ARCUS, while also establishing
partnerships to rapidly advance programs.”
Guest speakers will include:
- Mark Sulkowski, M.D. - Chief, Division of Infectious
Disease, John Hopkins University School of Medicine
- Carlos T. Moraes, Ph.D. - Lichtenstein Professor
for Neurology, University of Miami Miller School of Medicine
- Michio Hirano, M.D. - Chief, Division of Neuromuscular
Disorders, New York Presbyterian/Columbia University Irving Medical
Center
Highlighted R&D Day Topics
ARCUS Platform Overview
Precision will highlight key capabilities and differentiating
characteristics of its proprietary ARCUS genome editing platform,
which may enable ARCUS nucleases to drive more intended, defined
outcomes.
Pipeline Overview and Updates to Development Programs
- PBGENE-HBV is being developed for the treatment of
patients with chronic hepatitis B, with submission of a CTA and/or
IND application targeted for 2024. The Company will show data
highlighting the potential of PBGENE-HBV to eliminate covalently
closed circular DNA and inactivate integrated HBV DNA to drive
durable antigen loss, leading to a potential functional cure. In
addition, Dr. Alan List, Chief Medical officer at Precision, will
be joined by Dr. Mark Sulkowski for a fireside chat on the HBV
landscape.
- Precision will announce PBGENE-PMM as a potentially
first-in-class opportunity for treatment of m.3243 associated
primary mitochondrial myopathy. 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. The high specificity
and single component nature of Precision’s mitoARCUS nucleases are
designed to enable specific editing of mutant mitochondrial DNA
while allowing the normal (wild-type) mitochondrial DNA to
repopulate in the mitochondria and restore normal function.
Precision expects to submit a CTA and/or IND in 2025. The Company
will host a fireside chat between Dr. List, Dr. Carlos T. Moraes
and Dr. Michio Hirano to discuss the mitochondrial disease
space.
- PBGENE-NVS is being developed in collaboration with
Novartis for patients with hemoglobinopathies, such as sickle cell
disease and beta thalassemia. The companies intend to insert a
therapeutic transgene in vivo as a potential one-time
transformative treatment that could overcome many of the hurdles
present today with other therapeutic technologies, including those
that are utilizing an ex vivo gene editing approach.
- PBGENE-DMD is being developed with Prevail Therapeutics,
a wholly-owned subsidiary of Lilly, and is designed to utilize a
pair of ARCUS nucleases, delivered by a single AAV, to excise an
approximately 500,000 base pair mutation “hot spot” region from the
dystrophin gene, resulting in a variant of the dystrophin protein
that is functionally competent. The Company will highlight
preclinical data demonstrating the potential of ARCUS in vivo gene
editing for large gene excisions and that the edited dystrophin
variant was observed in multiple tissue types frequently involved
in progression of DMD, including skeletal muscle, heart, and
diaphragm, enabling significantly improved muscle function.
- iECURE-OTC is being developed by iECURE as a potential
treatment for neonatal onset ornithine transcarbamylase (OTC)
deficiency with a target CTA and/or IND submission in 2023. The
Company will highlight data demonstrating that iECURE-OTC showed
stable gene insertion at one year and that efficient targeted
insertion was achieved in non-human primates (NHPs) up to three
months of age. In addition, 12-month follow-up biopsies continued
to demonstrate durability, with gene insertion efficiency exceeding
20%, well above the expected threshold for clinical benefit.
Webcast Information
Registration for this virtual event and access to the live
webcast is available at http://precision2023rdday.q4ir.com or under
Events & Presentations in the Investors section of the
Precision BioSciences website at
https://investor.precisionbiosciences.com. The accompanying
presentation will also be available on the Events &
Presentations in the Investors section of the Precision BioSciences
website at https://investor.precisionbiosciences.com. The dial-in
conference call number is 1 (888) 800-8518. The conference ID
number for the call is 2795912. An archived replay of the webcast
will be available on the Company website for one year following the
presentation.
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. 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 of 1933,
as amended, and Section 21E of the Securities Exchange Act of 1934,
as amended. 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 clinical development and expected safety,
efficacy and benefit of our product candidates and gene editing
approaches including ARCUS’s potential editing efficiency and
differentiating aspects and the ability for the ARCUS genome
editing platform to develop differentiated programs; the
suitability of ARCUS nucleases for gene or viral elimination, large
excision, gene insertion, and other complex gene editing approaches
to drive defined outcomes; the expected timing of regulatory
processes; expectations about our operational initiatives and
business strategy; expectations around the Company’s partnerships;
and expectations about achievement of key milestones. The words
“aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,”
“designed”, “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 to advance our programs; risks
associated with raising additional capital and requirements under
our current debt instruments and effects of restrictions
thereunder; 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; the risk that other
genome-editing technologies may provide significant advantages over
our ARCUS technology; our dependence on our ARCUS technology; the
initiation, cost, timing, progress, achievement of milestones and
results of research and development activities and preclinical and
clinical studies; 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 product 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 or our collaborators’
ability to advance product candidates into, and successfully
design, implement and complete, clinical or field trials; 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; 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; the rate and degree of market
acceptance of any of our product candidates; our ability to
effectively manage the growth of our operations; our ability to
attract, retain, and motivate executives and personnel; effects of
system failures and security breaches; insurance expenses and
exposure to uninsured liabilities; effects of tax rules; effects of
any pandemic, epidemic, or outbreak of an infectious disease; 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; 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, 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 Securities and Exchange Commission
(“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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230912589832/en/
Investor and Media Contact: Mei Burris Director, Investor
Relations and Finance Mei.Burris@precisionbiosciences.com
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