- ARCUS Genome Editing Achieved Substantial
Reductions in both cccDNA and Hepatitis B Surface Antigen (HBsAg),
85% and 77% respectively, in HBV-infected Primary Human Hepatocytes
(PHH)
- Significant Decrease in HBsAg and High
On-target Editing Achieved in Novel Mouse and Non-human Primate
Models Following Lipid Nanoparticle (LNP) Delivery of ARCUS
mRNA
- Circulating HBsAg Surface Antigen was Durably
Decreased by 96% in Mice
- Data Published also Presented at the 2022
American Society of Gene and Cell Therapy (ASGCT) Annual
Meeting
- Precision to Continue Developing PBGENE-HBV
Product Candidate Using LNP Delivery and Expects to Submit an
IND/CTA in 2024
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage
gene editing company developing ARCUS-based ex vivo allogeneic CAR
T and in vivo gene editing therapies, today announced preclinical
research from its ongoing in vivo gene editing program targeting
hepatitis B virus (HBV) has been published online in Molecular
Therapy. Data from this study, “Targeting the
Hepatitis B cccDNA with a Sequence-Specific ARCUS Nuclease to
Eliminate Hepatitis B Virus In Vivo,” support the Company’s
continued development of its PBGENE-HBV candidate as a promising
gene editing approach that aims to eliminate virus persistence by
targeting covalently closed circular DNA (cccDNA) in patients with
chronic hepatitis B. This study was conducted in collaboration with
Gilead Sciences and Acuitas Therapeutics and was also presented
this week during the Gene Editing in Cancer and Complex Diseases
oral session at the ASGCT Annual Meeting.
“We’re very excited to see this study published in Molecular
Therapy and to showcase the compelling reductions in cccDNA and
surface antigen obtained with our ARCUS gene editing platform in
two novel animal models of HBV infection. Our data suggest that
LNP-delivered ARCUS mRNA is worth further exploration as a possible
functional cure for chronic hepatitis B,” said Derek Jantz, Chief
Scientific Officer and Co-founder of Precision BioSciences.
Precision’s gene editing program for chronic hepatitis B is
designed to apply ARCUS to knock out persistent cccDNA and
inactivate integrated hepatitis B genomes, potentially achieving
durable HBsAg loss and functional cure. In this preclinical study,
ARCUS efficiently targeted and degraded HBV cccDNA by 85% and
reduced expression of HBsAg by 77% in HBV-infected PHH.
Importantly, the optimized specificity of the ARCUS nuclease
completely prevented detectable chromosomal translocations in the
PHH model.
To evaluate ARCUS in vivo, novel mouse and non-human primate
models were developed that utilized an episomal adeno-associated
virus (AAV) containing a portion of the HBV genome to serve as a
surrogate for cccDNA. After administration of LNP containing ARCUS
mRNA, high on-target editing and a robust decrease in the cccDNA
surrogate was observed in both episomal models, along with a
durable 96% reduction of HBsAg in mice.
“The real challenge with HBV is that it persists in chronically
infected hepatocytes as an extrachromosomal genome called cccDNA.
Unless we can eliminate cccDNA, there’s always the potential that
the virus can reactivate,” continued Cassandra Gorsuch, Ph.D., Head
of Gene Therapy at Precision BioSciences and lead author of the
paper. “Current standard-of-care treatments for chronic hepatitis B
work by lowering or suppressing the amount of HBV found in infected
cells in the body but have no direct impact on the presence of
cccDNA, and therefore rarely clear the virus fully, necessitating
life-long therapy.”
Precision’s in vivo development pipeline currently comprises six
novel product candidates for genetic diseases. Three of these
product candidates are wholly owned -- PBGENE-HBV for chronic
hepatitis B, PBGENE-PH1 for primary hyperoxaluria type 1 and
PBGENE-PCSK9 for familial hypercholesteremia -- and Precision
expects to advance them to IND or CTA over the next three
years.
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 that was designed
with therapeutic safety, delivery and control in mind. Using ARCUS,
the Company’s pipeline consists of multiple ex vivo “off-the-shelf”
CAR T immunotherapy clinical candidates and several in vivo gene
editing candidates designed to cure 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 targeting hepatitis B cccDNA with ARCUS
nucleases in novel animal models. 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,”
“potential,” “predict,” “project,” “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. Such statements are subject to a 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
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 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 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; 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; 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 Annual
Report on Form 10-K for the fiscal year ended December 31 2021, as
any such factors may be updated from time to time in our other
filings with the SEC, including, but not limited to, our Quarterly
Report on Form 10-Q for the quarterly period ended March 31, 2022,
to be filed 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/20220518005451/en/
Investor Contact: Mei Burris Director, Finance and
Investor Relations Mei.Burris@precisionbiosciences.com
Media Contact: Maurissa Messier Senior Director,
Corporate Communications
Maurissa.Messier@precisionbiosciences.com
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