CRISPR Therapeutics Announces Preclinical Data at the American Heart Association (AHA) Scientific Sessions 2023
November 06 2023 - 5:01AM
CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company
focused on creating transformative gene-based medicines for serious
diseases, today announced preclinical data from the Company’s
investigational programs for the treatment of cardiovascular
disease, at the American Heart Association (AHA) Scientific
Sessions 2023. The data will be presented on Saturday, November 11,
2023, in two oral sessions, entitled “CTX310: An Investigational in
vivo CRISPR-Based Therapy Efficiently and Durably Reduces ANGPTL3
Protein and Triglyceride Levels in Non-Human Primates After a
Single Dose” and “CTX320: An Investigational in vivo CRISPR-Based
Therapy Efficiently and Durably Reduces Lipoprotein(a) Levels in
Non-Human Primates After a Single Dose."
“We’re excited to share these preclinical data
at AHA highlighting the progress made across our in vivo programs
targeting ANGPTL3 and Lp(a). The findings demonstrate the potential
of a one-time treatment to produce clinically meaningful,
long-lasting reductions in risk factors for atherosclerotic
cardiovascular disease,” said Phuong Khanh (P.K.) Morrow, M.D.,
FACP, Chief Medical Officer at CRISPR Therapeutics. “These data
increase our confidence in our goal to shift the treatment paradigm
for patients at risk of cardiovascular disease away from burdensome
chronic care to a potentially one-time, durable therapy.”
CRISPR Therapeutics is advancing a pipeline of
in vivo gene editing programs using lipid nanoparticle (LNP)
delivery of Cas9 mRNA and a guide RNA (gRNA) to the liver. The
first two in vivo programs, CTX310 and CTX320, each aim to reduce
expression of a validated target for cardiovascular disease.
CTX310 is an investigational in vivo CRISPR/Cas9
gene editing therapy designed to knock out hepatic expression of
angiopoietin-like 3 protein (ANGPTL3). In humans, naturally
occurring loss-of-function variants in ANGPTL3 are associated with
reduced levels of serum lipids and reduced risk of atherosclerotic
cardiovascular disease. In the preclinical data to be presented at
AHA, treatment of non-human primates (NHPs) with a single dose of
CTX310 led to mean editing of ANGPTL3 in the liver of 70%, and
corresponding mean reductions in plasma ANGPTL3 protein of more
than 85% and triglycerides of 60%. Reductions in ANGPTL3 protein
and triglycerides were durable past a year post-treatment. CTX310
was well-tolerated in NHPs with only transient elevation of liver
enzymes that resolved without intervention. These data suggest that
CTX310 has the potential to be used to treat dyslipidemias in
humans.
CTX320 is an investigational in vivo CRISPR/Cas9
gene editing therapy to reduce hepatic expression of lipoprotein(a)
(Lp(a)). Genetic and epidemiological studies in humans have
identified elevated levels of Lp(a) as an independent risk factor
of atherosclerosis and related diseases. In the preclinical data to
be presented at AHA, treatment of NHPs with a single dose of CTX320
led to a mean reduction in plasma Lp(a) of 95%. This reduction was
durable past a year post-treatment. CTX320 had a well-tolerated
safety profile similar to that of CTX310. These data suggest CTX320
has the potential to be used to reduce plasma Lp(a) levels in
humans.
About CRISPR Therapeutics
CRISPR Therapeutics is a leading gene editing
company focused on developing transformative gene-based medicines
for serious diseases using its proprietary CRISPR/Cas9 platform.
CRISPR/Cas9 is a revolutionary gene editing technology that allows
for precise, directed changes to genomic DNA. CRISPR Therapeutics
has established a portfolio of therapeutic programs across a broad
range of disease areas including hemoglobinopathies, oncology,
regenerative medicine and cardiometabolic diseases. To accelerate
and expand its efforts, CRISPR Therapeutics has established
strategic partnerships with leading companies including Bayer,
Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is
headquartered in Zug, Switzerland, with its wholly-owned U.S.
subsidiary, CRISPR Therapeutics, Inc., and R&D operations based
in Boston, Massachusetts and San Francisco, California, and
business offices in London, United Kingdom. For more information,
please visit www.crisprtx.com.
CRISPR THERAPEUTICS® standard character mark and
design logo, CTX310™ and CTX320™ are trademarks and registered
trademarks of CRISPR Therapeutics AG. All other trademarks and
registered trademarks are the property of their respective
owners.
CRISPR Therapeutics Forward-Looking
Statement
This press release may contain a number of
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995, as amended, including
statements made by Dr. Morrow in this press release, as well as
statements regarding CRISPR Therapeutics’ expectations about any or
all of the following: (i) the safety, efficacy and progress of
CRISPR Therapeutics’ various clinical and preclinical programs;
(ii) the status of clinical trials and preclinical studies
(including, without limitation, expectations regarding the oral
presentations, the data that is being presented, and the expected
timing of data releases and initiation of clinical trials); and
(iii) the therapeutic value, development, and commercial potential
of CRISPR/Cas9 gene editing technologies and therapies. Without
limiting the foregoing, the words “believes,” “anticipates,”
“plans,” “expects” and similar expressions are intended to identify
forward-looking statements. You are cautioned that forward-looking
statements are inherently uncertain. Although CRISPR Therapeutics
believes that such statements are based on reasonable assumptions
within the bounds of its knowledge of its business and operations,
forward-looking statements are neither promises nor guarantees and
they are necessarily subject to a high degree of uncertainty and
risk. Actual performance and results may differ materially from
those projected or suggested in the forward-looking statements due
to various risks and uncertainties. These risks and uncertainties
include, among others: uncertainties inherent in the initiation and
completion of preclinical studies for its product candidates and
whether results from such studies will be predictive of future
results of future studies or clinical trials; the potential that
clinical trial results may not be favorable or may not support
registration or further development; that one or more of its
clinical and preclinical programs will not proceed as planned for
technical, scientific or commercial reasons; the potential that
future competitive or other market factors may adversely affect the
commercial potential for CRISPR Therapeutics’ product candidates;
uncertainties regarding the intellectual property protection for
CRISPR Therapeutics’ technology and intellectual property belonging
to third parties; and those risks and uncertainties described under
the heading "Risk Factors" in CRISPR Therapeutics’ most recent
annual report on Form 10-K , quarterly report on Form 10-Q and in
any other subsequent filings made by CRISPR Therapeutics with the
U.S. Securities and Exchange Commission, which are available on the
SEC's website at www.sec.gov. CRISPR Therapeutics disclaims any
obligation or undertaking to update or revise any forward-looking
statements contained in this press release, other than to the
extent required by law.
Investor Contact:Susan
Kim+1-617-307-7503susan.kim@crisprtx.com
Media Contact:Rachel Eides
+1-617-315-4493rachel.eides@crisprtx.com
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