- High antiviral activity with substantial and
durable reductions in hepatitis B serum antigen and covalently
closed circular DNA in preclinical studies of PBGENE-HBV
- Supports continued PBGENE-HBV development and
planned submission of CTA/IND application
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage
gene editing company developing ARCUS®-based in vivo gene editing
and ex vivo allogeneic CAR T therapies, today announced that the
company will present positive preclinical data from its PBGENE-HBV
development program for the treatment of patients with chronic
hepatitis B during an oral presentation (Abstract #OS-034)
Thursday, June 22, 2023 at the European Association for Study of
the Liver (EASL) Congress 2023 in Vienna, Austria.
Hepatitis B virus (HBV) causes inflammation and damage to the
liver, leading to chronic infection and increased risk of death
from liver cancer or cirrhosis. There is no cure for chronic
hepatitis B and current treatments rarely result in functional
cure, primarily due to persistence of viral DNA in the liver. In
patients with chronic HBV, genetic material of the virus is
converted within infected liver cells into covalently closed
circular DNA (cccDNA) that acts as a template to make HBV copies.
HBV also inserts its DNA into the human genome of infected liver
cells. This integrated HBV DNA produces the viral protein,
hepatitis B surface antigen (HBsAg), which is secreted in the
blood. Presence of HBsAg is associated with poorer outcomes and
elimination of HBsAg is necessary for functional cure of chronic
hepatitis B. Using ARCUS, company scientists generated a highly
specific and effective nuclease to eradicate chronic HBV infection.
PBGENE-HBV is designed to inactivate cccDNA with direct cuts and
edits as well as to inactivate integrated HBV DNA leading to long
lasting reductions in HBsAg.
In an episomal adeno-associated virus (AAV) mouse model, company
researchers demonstrated that administration of lipid nanoparticles
containing mRNA encoding an HBV-targeted ARCUS nuclease resulted in
a 96% reduction in serum HBsAg. In a follow-on experiment,
treatment of HBV-infected primary human hepatocytes with the
HBV-targeted ARCUS nuclease resulted in a 90% reduction of
covalently closed circular DNA (cccDNA) and high specificity.
“Even with lifelong administration, the current treatments of
chronic HBV rarely achieve a functional cure because they do not
eradicate the cccDNA or inactivate the viral DNA that is integrated
into the human genome in hepatocytes. Eliminating both the cccDNA
and HBsAg are key parameters for achieving a functional cure of
HBV,” said Jeff Smith, Co-Founder and Chief Research Officer at
Precision Biosciences. “In preclinical models presented at EASL,
we’ve demonstrated that administration of HBV-targeted ARCUS
nucleases can achieve a near complete reduction in both HBsAg and
cccDNA.”
These data continue to highlight the potential of the ARCUS gene
editing platform to achieve a functional cure for patients and
support continued development of PBGENE-HBV with planned submission
of a CTA/IND application.
Presentation Details:
Title: A Gene Editing Approach for Chronic Hepatitis B:
Elimination of Hepatitis B Virus In Vivo by Targeting cccDNA and
Integrated Viral Genomes with a Sequence-Specific ARCUS Nuclease
Authors: Gorsuch C, et al. Date and Time: Thursday,
June 22, 2023, 6:15-6:30pm CEST / 12:15-12:30pm EDT Abstract
Number: OS-034 Session Title: Abstract session - Viral
hepatitis B/D - New treatments Session Room: Strauss 2-3
About Hepatitis B and the PBGENE-HBV development
candidate:
Hepatitis B is a leading cause of morbidity in the US and death
globally, with no curative options currently available for
patients. In 2019, despite the availability of approved antiviral
therapies, an estimated 300 million people globally and more than 1
million people in the US were estimated to have chronic hepatitis B
infection. An estimated 15% to 40% of patients with HBV infections
may develop complications, such as cirrhosis, liver failure, or
liver cancer (hepatocellular carcinoma), which account for the
majority of HBV-related deaths.
Chronic hepatitis B infection is primarily driven by persistence
of HBV cccDNA and integration of HBV DNA into the human genome in
liver cells, the primary source of HBsAg in late-stage disease.
Current treatments for patients with HBV infection include agents
that result in long-term viral suppression as indicated by
reduction of circulating HBV DNA, but these therapies do not
eradicate HBV cccDNA, rarely lead to functional cure, and require
lifelong administration. PBGENE-HBV is a highly specific, novel
therapeutic approach to treating patients with chronic HBV
infection. It’s designed to directly cut and edit cccDNA and
inactivate integrated HBV DNA with high specificity, resulting in
degraded cccDNA and a reduction in HBsAg.
About Precision BioSciences, Inc.
Precision BioSciences, Inc. is a clinical stage biotechnology
company dedicated to improving life (DTIL) with its novel and
proprietary ARCUS® genome editing platform. ARCUS is a highly
precise and versatile genome editing platform designed with
therapeutic safety, delivery, and control in mind. Using ARCUS, the
Company’s pipeline consists of several in vivo gene editing
candidates designed to cure genetic and infectious diseases where
no adequate treatments exist and multiple ex vivo clinical
candidates. 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 expected safety, efficacy, and benefit of
our gene editing approaches including editing efficiency and
delivery methods, the suitability of ARCUS nucleases for gene
excision, the clinical development, nomination, and goals of our
PBGENE-HBV program, the timing of the submission of a CTA/IND
application for PBGENE-HBV, and therapeutic potential of an ARCUS
gene editing approach for the treatment of chronic hepatitis B. 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. 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; 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; potential manufacturing problems associated with the
development or commercialization of any of our product candidates;
our ability to obtain an adequate supply of T cells from qualified
donors; our ability to achieve our anticipated operating
efficiencies at our manufacturing facility; 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 the COVID-19 pandemic and variants
thereof, or 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
March 31, 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/20230622589961/en/
Investor and Media Contact: Mei Burris Director, Investor
Relations and Finance Mei.Burris@precisionbiosciences.com
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