Late-breaking, oral presentation highlights
ability of ARCUS gene editing approach to achieve large gene
excisions enabling significant functional muscle improvement in
preclinical study
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 preclinical data demonstrating the potential
of ARCUS in vivo gene editing for large gene excisions toward the
goal of treating Duchenne muscular dystrophy (DMD) at the American
Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting. The
oral presentation, titled “ARCUS-Mediated Excision of the “Hot
Spot” Region of the Human Dystrophin Gene for the Treatment of
Duchenne Muscular Dystrophy (DMD),” will be delivered during the
Late-breaking Abstracts 2 Session held today from 10:15 AM to 12:00
PM PT.
“While traditional gene therapies have shown promise in
potentially slowing or stabilizing the progression of DMD, there
remains no curative therapy for this disorder,” said Jeff Smith,
Chief Research Officer of Precision BioSciences. “During today’s
presentation, we are excited to share the first in vivo data
demonstrating the therapeutic potential of an ARCUS gene editing
approach for DMD, which may one day enable the single
administration of a drug with life-long benefits of muscle
retention and function to a broad patient population.”
Precision’s PBGENE-DMD program strategy is to restore expression
of a functional form of dystrophin by utilizing a pair of ARCUS
nucleases that are delivered by a single adeno-associated virus
(AAV) to excise an approximately 500,000 base pair mutation “hot
spot” region of the dystrophin gene, resulting in a variant of the
dystrophin protein that is functionally competent. Up to 50% of DMD
patients have pathogenic mutations in this region, suggesting this
editing strategy could have broad applicability compared to
mutation-specific approaches.
In the data reported today using early generation ARCUS
nucleases, scientists observed the edited dystrophin variant in
multiple tissue types frequently involved in progression of DMD,
including skeletal muscle, heart, and diaphragm. Furthermore, the
maximum force output of the gastrocnemius muscle in ARCUS-treated
animals was significantly improved compared to untreated mice,
reaching 86% of the maximum force output levels observed in
non-diseased, control animals.
“We believe that Precision’s approach to DMD is differentiated
by the potential of ARCUS nucleases to precisely excise large
genomic regions and repair the gene with high efficiency,” said
Cassie Gorsuch, VP of Gene Therapy at Precision. “ARCUS nucleases
have the unique capability to generate 4 base pair 3’ overhangs, or
‘sticky ends,’ following DNA cleavage. In our PBGENE-DMD program,
we’ve engineered a pair of ARCUS nucleases that generate
complementary overhangs at their target sites in the dystrophin
gene to promote perfect re-ligation after excision of the ‘hot
spot’ region. Due to the small size of ARCUS nucleases, we are able
to deliver both using a single AAV.” The in vivo proof-of-concept
study presented today demonstrates the therapeutic potential of an
ARCUS gene editing approach for the treatment of DMD and highlights
the unique advantages of the ARCUS gene editing platform.
About Duchenne muscular dystrophy DMD is a genetic
disorder associated with mutations in the dystrophin gene that
prevent production of the dystrophin protein. Dystrophin stabilizes
the cell membrane during muscle contraction to prevent damage, and
the absence of intact dystrophin protein leads to inflammation,
fibrosis, and progressive loss of muscle function and mass. Over
time, children with DMD will develop problems walking and
breathing, eventually leading to death in the second or third
decade of life due to progressive cardiomyopathy and respiratory
insufficiency. DMD occurs in 1 in 3,500 to 5,000 male births, and
currently there are limited approved therapies available for
patients.
About ARCUS ARCUS is a proprietary genome editing
technology discovered and developed by scientists at Precision
BioSciences. It uses sequence-specific DNA-cutting enzymes, or
nucleases, that are designed to either insert (knock-in), excise
(knock-out), or repair DNA of living cells and organisms. ARCUS is
based on a naturally occurring genome editing enzyme, I-CreI, that
evolved in the algae Chlamydomonas reinhardtii to make highly
specific cuts in cellular DNA and stimulate gene insertion at the
cut site by homologous recombination. Precision's platform and
products are protected by a comprehensive portfolio including
nearly 100 patents to date.
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 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 expected conference
participation and disclosure of preclinical data, the clinical
development, nomination, and goals of our PBGENE-DMD program,
therapeutic potential of an ARCUS gene editing approach for the
treatment of DMD, and expected safety, efficacy, and benefit of our
gene editing approaches including re-ligation editing efficiency.
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period ended March 31, 2023, as any such factors may be updated
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version on businesswire.com: https://www.businesswire.com/news/home/20230519005059/en/
Investor and Media Contact: Mei Burris Director, Investor
Relations and Finance Mei.Burris@precisionbiosciences.com
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