- OTC Deficiency Gene Insertion Program,
ECUR-506, Receives Clearance from Australian Therapeutic Goods
Administration for Phase 1/2 Study
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
Australian Therapeutic Goods Administration for the initiation of a
first-in-human Phase 1/2 trial evaluating ECUR-506, incorporating
an ARCUS nuclease for the treatment of Ornithine Transcarbamylase
(OTC) deficiency in pediatric (or neonatal) patients.
“The acceptance of iECURE’s Clinical Trial Application marks an
important milestone for patients with OTC deficiency and for both
the iECURE and Precision BioSciences teams. This is the first ARCUS
in vivo gene editing program to progress into the clinic,” said
Michael Amoroso, Chief Executive Officer of Precision Biosciences.
“We look forward to supporting iECURE's continued progress with
this program while working expeditiously to advance our
wholly-owned hepatitis B virus program towards an anticipated
clinical trial application (CTA) and/or investigational new drug
(IND) 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.
In preclinical studies, administration of ECUR-506 resulted in a
durable response in nonhuman primates, with up to 28.2% of liver
cells demonstrating expression of the human OTC gene at the 1-year
time point. A 5% threshold of enzymatic activity has the potential
for clinical benefit as previously identified in Annals of Clinical
and Translational Neurology. Separately, in a mouse model of
OTC-deficiency, the injection of ECUR-506 in newborn mice
efficiently inserted a healthy copy of the OTC gene.
“At Precision, we have taken a methodical approach in the
development, optimization, and validation of ARCUS as a
differentiated tool that is uniquely suited for high efficiency
gene insertion due to its unique cut which drives homology directed
repair (HDR). As presented in preclinical work at the European
Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress
earlier this year, current ARCUS nucleases demonstrated 17 times
higher gene insertion efficiency in primary human T-cells compared
to CRISPR-Cas9. In addition, ARCUS showed up to approximately 45%
insertion when administered by lipid nanoparticle along with an
adeno-associated virus carrying a gene insertion template in adult
non-human primates, and demonstrated high efficiency gene insertion
via HDR in nondividing, primary human hepatocytes. Today’s
announcement of a regulatory clearance for human clinical trials
further validates our ARCUS approach and highlights its potential
to deliver a curative treatment for patients with OTC deficiency,”
said Jeff Smith, Ph.D., Chief Research Officer at Precision
BioSciences. “ECUR-506 is the first of several partnered and wholly
owned gene editing programs leveraging ARCUS in differentiated
indications and we look forward to initial clinical data from this
program.”
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.
About iECURE
iECURE is a 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. iECURE’s 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. iECURE is 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. For more information, visit iecure.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 potential of an ARCUS gene editing
approach as a curative treatment for OTC deficiency, including
durability of response, anticipated timing of a CTA and/or IND
filing for wholly-owned programs, the ability and differentiation
of ARCUS for high efficiency gene insertion and driving homology
directed repair, expected safety, efficacy, and benefit of our gene
editing approaches, and anticipated timing of initial clinical
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Forward-looking statements are based on management’s current
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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
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and efficacy of our product candidates; our dependence on our ARCUS
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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
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
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version on businesswire.com: https://www.businesswire.com/news/home/20231212100358/en/
Investor and Media Contact: Mei Burris Senior Director of
Finance and Controller Mei.Burris@precisionbiosciences.com
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