Intellia Therapeutics Presents Preclinical Proof of Concept for CRISPR-based In Vivo Editing of Bone Marrow at Keystone eSymp...
March 10 2021 - 6:30AM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), today announced the
presentation of preclinical data establishing proof-of-concept for
non-viral genome editing of bone marrow and hematopoietic stem
cells (HSCs) in mice. This represents the company’s first
demonstration of systemic in vivo genome editing in tissue outside
the liver using its proprietary non-viral delivery platform. Gene
editing of HSCs in vivo via a non-viral delivery system offers the
potential to transform the treatment of sickle cell disease (SCD)
and other inherited blood disorders by overcoming the complexity
and safety risks of ex vivo approaches. The company is presenting
these data today at the Keystone eSymposium: Precision Engineering
of the Genome, Epigenome and Transcriptome, being held virtually
March 8-10, 2021.
“This new data supports the possibility of delivering a safer
solution to treat blood disorders, including sickle cell disease,
by avoiding the need for bone marrow transplantation,” said
President and Chief Executive Officer, John Leonard, M.D. “We’ve
demonstrated we can expand our in vivo capabilities originally
designed for liver applications to other tissues and achieve
therapeutically meaningful levels of gene editing, reinforcing the
promise of Intellia’s modular platform to transform the lives of
people living with genetic diseases.”
Presentation DetailsTitle: “In
Vivo Genome Editing of Hematopoietic Stem and Progenitor
Cells”Session: DeliveryDate and
Time: March 10, 2021, 11:50 a.m. - 12:05 p.m.
ETPresenting Author: Sean Burns M.D., senior
director of Intellia’s Disease Biology and Pharmacology group
The presentation can be found here, on the Scientific
Publications & Presentations page of Intellia’s website.
CRISPR/Cas9-based genome editing is well suited to the treatment
of hereditary blood disorders, such as SCD. However, the current
requirement for ex vivo manipulation of HSCs and toxic
myeloablative transplantation regimens are significant barriers to
widespread adoption of this approach. An in vivo gene editing
strategy, in which CRISPR/Cas9 is delivered systemically as a
treatment into patients, could greatly reduce the risk, cost and
barriers to treatment associated with ex vivo genomic modification
of HSCs. Such an approach could provide a one-time, curative
treatment option for patients worldwide who are suffering from
SCD.
About the StudyIntellia’s non-viral delivery
platform enables systemic administration of CRISPR/Cas9 to
disease-relevant tissues. The findings being presented today
demonstrate the applicability of this platform for editing cells
within the bone marrow. In this proof of concept study:
- Lipid nanoparticles (LNPs) enabled transient and well-tolerated
delivery of CRISPR/Cas9 to murine and human hematopoietic stem and
progenitor cells (HSPCs) in mice
- Dose-dependent editing was seen in whole bone marrow as well as
HSPCs, with editing levels in hematopoietic stem cells found to be
durable for over one year after a single LNP administration
- Editing increased upon repeat LNP administration, potentially
enabling a “treat-to-target” approach
- Transplantation studies showed that in vivo edited HSPCs
retained their capacity to provide long term, multilineage
reconstitution of bone marrow
- In vivo editing in a humanized mouse model demonstrated
relevance of the approach to human HSPCs
Intellia is currently building upon this preclinical work,
towards a potential cure for SCD, with a grant from the Bill &
Melinda Gates Foundation.
About Sickle Cell DiseaseSickle cell disease
(SCD) affects >100,000 people in the U.S. and millions of people
worldwide, with highest prevalence in sub-Saharan Africa and India.
SCD is caused by a mutation in the beta globin gene that leads to
sickling of red blood cells (RBCs) and clotting in small arteries.
The disease is characterized by severe pain and multi-organ injury,
including in the brain, heart, lungs, kidneys and joints, with
greatly reduced life expectancy and quality of life. Standard of
care therapies include hydroxyurea and frequent RBC transfusions
from healthy donors. Allogenic hematopoietic stem cell
transplantation is reserved as a last resort for severely affected
patients, though is limited by associated morbidities, high cost
and the need for specialized clinical care, and is generally not
accessible to patients in countries with limited healthcare
resources.
About Intellia TherapeuticsIntellia
Therapeutics is a leading clinical-stage genome editing company,
focused on the development of proprietary, potentially curative
therapeutics using the CRISPR/Cas9 system. Intellia believes the
CRISPR/Cas9 technology has the potential to transform medicine by
both producing therapeutics that permanently edit and/or correct
disease-associated genes in the human body with a single treatment
course, and creating enhanced engineered cells that can treat
oncological and immunological diseases. Intellia’s combination of
deep scientific, technical and clinical development experience,
along with its leading intellectual property portfolio, puts it in
a unique position to unlock broad therapeutic applications of the
CRISPR/Cas9 technology and create new classes of therapeutic
products. Learn more about Intellia and CRISPR/Cas9 at
intelliatx.com. Follow us on Twitter @intelliatweets.
Forward-Looking StatementsThis press release
contains “forward-looking statements” of Intellia Therapeutics,
Inc. (“Intellia”, “we” or “our”) within the meaning of the Private
Securities Litigation Reform Act of 1995. These forward-looking
statements include, but are not limited to, express or implied
statements regarding Intellia’s beliefs and expectations regarding
our: plans to advance and complete preclinical studies for our
programs, including non-viral extrahepatic delivery to bone marrow
to treat sickle cell disease (“SCD”) and other hemopoietic stem
cell (HSC)-inherited disorders; development of our modular platform
to advance our complex genome editing capabilities; advancement and
expansion of our CRISPR/Cas9 technology to develop human
therapeutic products, as well as our ability to maintain and expand
our related intellectual property portfolio; ability to demonstrate
our platform’s modularity and replicate or apply results achieved
in preclinical studies, including those in our SCD and other
HSC-inherited programs, in any future studies, including human
clinical trials; ability to develop other in vivo or ex vivo cell
therapeutics of all types, and those targeting SCD and other
HSC-inherited diseases in particular, using CRISPR/Cas9 technology;
ability to expand, maintain and protect our intellectual property
rights, including patents and licenses; and potential commercial
opportunities, including value and market, for our product
candidates.
Any forward-looking statements in this press release are based
on management’s current expectations and beliefs of future events,
and are subject to a number of risks and uncertainties that could
cause actual results to differ materially and adversely from those
set forth in or implied by such forward-looking statements. These
risks and uncertainties include, but are not limited to: risks
related to our ability to protect and maintain our intellectual
property position; risks related to our relationship with third
parties, including our licensors and licensees; risks related to
the ability of our licensors to protect and maintain their
intellectual property position; uncertainties related to regulatory
agencies’ evaluation of regulatory filings and other information
related to our product candidates; uncertainties related to the
authorization, initiation and conduct of studies and other
development requirements for our product candidates; the risk that
any one or more of our product candidates, including those that are
co-developed, will not be successfully developed and
commercialized; and the risk that the results of preclinical
studies or clinical studies will not be predictive of future
results in connection with future studies. For a discussion of
these and other risks and uncertainties, and other important
factors, any of which could cause Intellia’s actual results to
differ from those contained in the forward-looking statements, see
the section entitled “Risk Factors” in Intellia’s most recent
annual report on Form 10-K as well as discussions of potential
risks, uncertainties, and other important factors in Intellia’s
other filings with the Securities and Exchange Commission (“SEC”).
All information in this press release is as of the date of the
release, and Intellia undertakes no duty to update this information
unless required by law.
Intellia Contacts:
Investors:Glenn GoddardChief Financial
Officer+1-857-706-1056glenn.goddard@intelliatx.com
Media:Julie Ferguson Interim Head, External
Affairs &
CommunicationsJulie.ferguson@intelliatx.com
+1-312-385-0098
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