- Insertion of a functional OTC gene through
ARCUS in vivo gene editing may provide lasting clinical benefit for
children with OTC deficiency who are in dire need for effective
treatments
- CTA approval for ECUR-506 in the United
Kingdom provides further regulatory support for Precision
BioSciences’ ARCUS in vivo gene editing platform
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 that its partner iECURE has received approval from the
U.K. Medicines & Healthcare products Regulatory Agency (MHRA)
for the company’s Clinical Trial Authorization (CTA) application to
expand the Phase 1/2 OTC-HOPE study evaluating ECUR-506 into the
U.K. The OTC-HOPE study is investigating ECUR-506, incorporating an
ARCUS nuclease, for the treatment of Ornithine Transcarbamylase
(OTC) deficiency in infants. The CTA approval by the MHRA follows
the previous approval to begin the OTC-HOPE study by the Australian
Therapeutic Goods Administration (TGA). In 2021, Precision licensed
to iECURE an ARCUS nuclease that inserts a functional copy of the
OTC gene for treatment of OTC deficiency.
“Congratulations to iECURE for bringing the first gene editing
approach into clinical investigation for patients with OTC
deficiency. Approval from the MHRA to expand iECURE’s OTC-HOPE
Phase 1/2 study represents yet another milestone, which both
advances our partner’s work to potentially revolutionize the
treatment of OTC deficiency in infants while validating the
differentiated ability of ARCUS to efficiently insert genes and
restore function,” said Michael Amoroso, Chief Executive Officer of
Precision BioSciences. “To that end, we view progression through
multiple regulatory agencies around the world as important
proof-points for ARCUS that reflects our broader strategy to
leverage ARCUS with select development partners as we continue to
advance our wholly owned hepatitis B program toward an
investigational new drug (IND) application and/or CTA filing in
2024.”
OTC deficiency, the most common urea cycle disorder, is an
inherited metabolic disorder caused by a genetic defect in a liver
enzyme responsible for the detoxification of ammonia. Individuals
with OTC deficiency can build up excessive levels of ammonia in
their blood potentially resulting in devastating consequences,
including irreversible neurological damage, coma, and death. The
severe form of the condition emerges shortly after birth and is
more common in boys than girls. The only corrective treatment for
early onset severe OTC deficiency is a liver transplant. Currently
available medical therapies do not correct the disease and do not
eliminate the risk of life-threatening symptoms or crises.
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, Precision’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.
About ECUR-506
iECURE’s approach to gene editing for its initial programs,
including OTC deficiency, relies on the delivery of two
adeno-associated virus (AAV) capsids, each carrying different
payloads. ECUR-506 comprises two vectors, an ARCUS® nuclease vector
targeting gene editing in the well-characterized PCSK9 gene locus
and a donor vector that inserts the desired functional OTC gene.
iECURE has licensed the ARCUS nuclease for ECUR-506 from Precision
BioSciences. The cut in the PCSK9 site serves as the insertion site
for the OTC gene, providing a potential path to permanent
expression of a healthy gene.
About the OTC-HOPE Study
The OTC-HOPE study is a Phase 1/2 first-in-human clinical trial
of ECUR-506 in baby boys with genetically confirmed OTC deficiency
and will test up to two dose levels of ECUR-506. The study is
enrolling baby boys aged 24 hours to seven months who are diagnosed
with severe neonatal onset OTC deficiency and meet certain other
criteria. The primary objective is to assess the safety and
tolerability of intravenous administration of a single dose of
ECUR-506. It will also assess the pharmacokinetics and efficacy of
ECUR-506 administration and the potential effects of ECUR-506 on
disease-specific biologic markers, developmental milestones and
quality of life.
About iECURE
iECURE is a clinical-stage gene editing company focused on
developing therapies that utilize mutation-agnostic in vivo gene
insertion, or knock-in, editing for the treatment of liver
disorders with significant unmet need. We believe our approach has
the potential to replace and restore the function of a
dysfunctional gene, regardless of mutation, by knocking-in a
healthy copy of that gene to offer durable gene expression and
long-term, potentially curative, therapeutic benefit. Our
management team has extensive experience in executing global orphan
drug and gene therapy clinical trials and successfully
commercializing multiple products. We intend to leverage our team’s
core strength in research and development strategy to identify what
we believe to be the most suitable target and modality for our
product candidates to address particular liver diseases. We are
collaborating with the University of Pennsylvania’s Gene Therapy
Program (GTP) led by James M. Wilson, M.D., Ph.D., to utilize GTP’s
world-class translational expertise and infrastructure, which has
helped generate our initial pipeline of potential product
candidates. For more information, visit https://iecure.com and
follow on LinkedIn.
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 and our partners’ product candidates
and gene editing approaches including editing efficiency; the
suitability of ARCUS nucleases for gene insertion and other gene
editing approaches; the expected timing of regulatory processes;
expectations about our and our partners’ operational initiatives
and strategy; 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,”
“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, and involve 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 to advance our programs; risks
associated with our capital requirements, anticipated cash runway,
requirements under our current debt instruments and effects of
restrictions thereunder, including our ability to raise additional
capital due to market conditions and/or our market capitalization;
our operating expenses and our ability to predict what those
expenses will be; our limited operating history; the progression
and 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, including clinical trial and
investigational new drug applications; public perception about
genome editing technology and its applications; competition in the
genome editing, biopharmaceutical, and biotechnology fields; our or
our collaborators’ or other licensees’ ability to identify, develop
and commercialize product candidates; pending and potential product
liability lawsuits and penalties against us or our collaborators or
other licensees related to our technology and our product
candidates; the U.S. and foreign regulatory landscape applicable to
our and our collaborators’ or other licensees’ 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; 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; delays
or difficulties in our and our collaborators’ and other licensees’
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 or our
licensees’ 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’ or other
licensees’ 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; 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
September 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/20240306169732/en/
Naresh Tanna Vice President of Investor Relations
Naresh.Tanna@precisionbiosciences.com
Precision BioSciences (NASDAQ:DTIL)
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