Intellia Therapeutics Receives Grant to Develop Curative CRISPR/Cas9 In Vivo Sickle Cell Disease Treatments
11 November 2020 - 11:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), announced that it has
received a grant from the Bill & Melinda Gates Foundation to
research in vivo sickle cell disease (SCD) treatments using its
CRISPR/Cas9 genome editing technology. This pilot development
program is part of the Gates Foundation’s broader initiative to
accelerate the advancement of safe, effective and durable
gene-based cures in developing countries within the next seven to
10 years. The funding from the Gates Foundation will advance
Intellia’s preclinical validation of in vivo hematopoietic stem
cells (HSCs) genome editing using the company’s proprietary
non-viral delivery systems and CRISPR/Cas9 technology to
potentially cure SCD.
“We are excited to receive funding from the Gates Foundation to
take the first steps toward development of a potential in vivo
non-viral CRISPR/Cas9-based cure for SCD. Genome editing
offers multiple opportunities to treat SCD as shown by encouraging
emerging clinical data. Our goal is to deliver on the already
demonstrated promise of CRISPR/Cas9, but avoid the severe
complications of bone marrow transplantation that may limit the
usefulness of current approaches,” said Intellia’s Chief Scientific
Officer Laura Sepp-Lorenzino, Ph.D. “Intellia’s ambition is to
use our non-viral in vivo platform to create an
innovative treatment for blood disorders, that is scalable and can
overcome the challenges inherent to ex vivo cell-based
therapies for global diseases.”
Intellia’s in vivo CRISPR/Cas9 delivery approach has
the potential to develop HSC-based genome editing therapies that
are more practical solutions to treat blood disorders, including
SCD. Intellia’s in vivo platform technology delivers
CRISPR/Cas9 intravenously, potentially avoiding the need for bone
marrow transplantation surgery.
About Sickle Cell DiseaseSickle cell disease
(SCD) affects millions of people throughout the world and is
particularly common among those whose ancestors came from
sub-Saharan Africa, certain regions in South and Central America,
Saudi Arabia, India and some Mediterranean countries. SCD
disproportionately affects populations in sub-Saharan Africa and
India and leads to substantial morbidity, mortality and economic
disadvantages. In the United States, approximately 100,000 people
have been diagnosed, whereas in Nigeria, the country with the
highest known incidence of SCD, there are approximately 150,000
babies born annually with sickle cell anemia.
About Intellia TherapeuticsIntellia
Therapeutics is a leading 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 Therapeutics and
CRISPR/Cas9 at intelliatx.com. Follow us on Twitter
@intelliatweets.
Forward-Looking Statements
This press release contains “forward-looking statements”
of Intellia Therapeutics, Inc. (“Intellia” or the
“Company”) 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 its: advancement and
expansion of its CRISPR/Cas9 technology to develop human
therapeutic products, as well as its ability to maintain and expand
its related intellectual property portfolio; ability to demonstrate
its platform’s modularity and replicate or apply results achieved
in preclinical studies in any future studies, including human
clinical trials; expectations of the potential impact of the
coronavirus disease 2019 pandemic on strategy, future operations
and timing of its clinical trials or IND submissions; ability to
optimize the impact of its collaborations on its development
programs; use of capital, expenses, future accumulated deficit and
other 2020 financial results or in the future; and ability to fund
operations for at least the next 24 months.
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 Intellia’s ability to protect and maintain its
intellectual property position; risks related to Intellia’s
relationship with third parties, including its licensors and
licensees; risks related to the ability of its licensors to protect
and maintain their intellectual property position; uncertainties
related to the authorization, initiation and conduct of studies and
other development requirements for its product candidates; the risk
that any one or more of Intellia’s product candidates will not be
successfully developed and commercialized; the risk that the
results of preclinical studies or clinical studies will not be
predictive of future results in connection with future studies; and
the risk that Intellia’s collaborations or grants will not continue
or will not be successful. 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. 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:
Media:Jennifer Mound SmoterSenior Vice
PresidentExternal Affairs & Communications+1
857-706-1071jenn.smoter@intelliatx.com
Lynnea OlivarezDirectorExternal Affairs & Communications+1
956-330-1917lynnea.olivarez@intelliatx.com
Investors:Lina LiAssociate DirectorInvestor
Relations+1 857-706-1612lina.li@intelliatx.com
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