- PBGENE-HBV specifically cuts HBV DNA without
impacting the human genome
- PBGENE-HBV was well tolerated across multiple
administrations with no off-target editing observed
- Preclinical safety data supports advancement
of PBGENE-HBV to clinical trials as a potentially curative, finite
treatment for chronic hepatitis B with IND and/or CTA on-track for
submission in 2024
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 will present preclinical data for its PBGENE-HBV clinical
candidate at the European Association for the Study of the Liver
Congress (EASL), highlighting the differentiated ability of ARCUS
to make efficient, durable, and targeted elimination edits. The
poster presentation highlights data in primary human hepatocytes
demonstrating the high specificity and lack of detectable
off-target editing for PBGENE-HBV at therapeutically relevant
doses. The poster will also showcase non-human primate data
demonstrating good tolerability of a multi-dosing approach for the
treatment of chronic hepatitis B.
“We are pleased to present new safety data at EASL for our lead
PBGENE-HBV program. These data highlight our ability to
specifically target the HBV viral genome, with no detectable
off-target editing in the human genome at relevant doses. In
addition, the new non-human primate data shows that multiple
administrations of PBGENE-HBV are well tolerated, enabling us to
multi-dose in clinic,” said Jeff Smith, PhD, Chief Research Officer
of Precision BioSciences. “Our work to date continues to highlight
the potential of PBGENE-HBV as a curative treatment for chronic
hepatitis B and further validates the capabilities of our
proprietary ARCUS® platform to deliver safe and efficacious in vivo
gene editing therapies. Looking ahead, we are well positioned to
progress PBGENE-HBV, our first wholly owned ARCUS program into the
clinic with an Investigational New Drug Application (IND) and/or
Clinical Trial Application (CTA) in the 2024.”
The data to be presented highlights that PBGENE-HBV specifically
cuts HBV DNA leading to elimination of cccDNA and inactivation of
integrated HBV DNA without impacting any sites in the human genome,
including no editing-associated translocations in HBV infected
primary human hepatocytes. In addition, PBGENE-HBV was
well-tolerated in non-human primates across multiple dose
administrations, with only minor and transient elevations in liver
transaminases that are normalized within 2 weeks and non-adverse
changes in blood parameters. Preclinical safety data supports the
advancement of PBGENE-HBV to clinical trials as a potentially
curative treatment for chronic hepatitis B.
Full poster details are included below:
Title: Preclinical safety data for PBGENE-HBV gene
editing program supports advancement to clinical trials as a
potentially curative treatment for chronic hepatitis B Abstract
Number: LB195 Presenter: Emily Harrison, PhD, Senior
Scientist - Hepatitis Research Leader, Precision BioSciences
Date and Time: Wednesday, June 5, 2024, 8:30 AM – Saturday,
June 8, 2024, 5:00 PM CEST
About Hepatitis B and PBGENE-HBV:
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 eliminate cccDNA and
inactivate integrated HBV DNA with high specificity, potentially
leading to functional cures.
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.
The ARCUS® platform is being used to develop in vivo gene
editing therapies for sophisticated gene edits, including gene
insertion (inserting DNA into gene to cause expression/add
function), elimination (removing a genome e.g. viral DNA or mutant
mitochondrial DNA), and excision (removing a large portion of a
defective gene by delivering two ARCUS nucleases in a single
AAV).
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 clinical development and expected safety,
efficacy and benefit of our product candidates and gene editing
approaches including editing efficiency; the design of PBGENE-HBV
to directly eliminate cccDNA and inactivate integrated HBV DNA with
high specificity, potentially leading to functional cures; the
suitability of ARCUS nucleases for gene elimination, insertion and
excision and differentiation from other gene editing approaches due
to its small size, simplicity and distinctive cut; the expected
timing of regulatory processes (including filings such as IND’s and
CTA’s and studies for PBGENE-HBV); expectations about operational
initiatives, strategies, and further development of our programs;
expectations about achievement of key milestones; and anticipated
timing of clinical data. In some cases, you can identify
forward-looking statements by terms such as “aim,” “anticipate,”
“approach,” “believe,” “contemplate,” “could,” “designed,”
“estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,”
“plan,” “possible,” “potential,” “predict,” “project,” “pursue,”
“should,” “strive,” “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 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; 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; 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’ or other licensees’
ability to advance product candidates into, and successfully
design, implement and complete, clinical or field trials; our or
our collaborators’ other licensees’ 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; 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 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; 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
March 31, 2024, 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
presentation 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. Precision
consults with various presentation speakers and compensates them
for their time and expertise.
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version on businesswire.com: https://www.businesswire.com/news/home/20240605698770/en/
Investor and Media Contact: Naresh Tanna Vice President
of Investor Relations naresh.tanna@precisionbiosciences.com
Precision BioSciences (NASDAQ:DTIL)
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