Intellia Therapeutics’ Investigational CRISPR Treatment NTLA-2001 Receives European Union Orphan Drug Designation for ATTR ...
30 Mars 2021 - 1:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on developing curative therapeutics using
CRISPR/Cas9 technology both in vivo and ex vivo, announced today
that the European Commission (EC) has granted orphan drug
designation to NTLA-2001. This investigational medicinal product is
being developed as a treatment for transthyretin amyloidosis
(ATTR), a rare condition that can impact a number of organs and
tissues within the body through the accumulation of misfolded
transthyretin (TTR) protein deposits. NTLA-2001 is the first
experimental CRISPR therapy – a Nobel Prize-winning technology – to
be administered systemically via intravenous dosing to edit a gene,
specifically the TTR gene, that encodes the production of TTR
protein, inside the human body. In addition to being the first
therapy of its kind, it also has the potential to be the first
curative treatment and it may be able to halt and reverse ATTR
progression.
“This news is a significant milestone for NTLA-2001 and the ATTR
patient community,” said Intellia President and Chief Executive
Officer John Leonard, M.D. “We are pleased that the EC recognizes
the potential significant benefit of NTLA-2001 in the treatment of
patients with this debilitating disease where there is no cure. We
look forward to advancing the global development of this genome
editing product in collaboration with Regeneron.”
This decision by the EC follows the initiation of Intellia’s
global Phase 1 study to evaluate NTLA-2001 for hereditary ATTR with
polyneuropathy (hATTR-PN). The trial aims to assess the safety,
tolerability, pharmacokinetics and pharmacodynamics of NTLA-2001.
Following safety assessment and dose optimization, Intellia intends
to further evaluate NTLA-2001 in both polyneuropathy and
cardiomyopathy patients. Orphan drug designation is granted to
therapies that are intended for the treatment, prevention, or
diagnosis of life threatening or chronically debilitating rare
diseases where there are either no treatments or no satisfactory
therapeutic options. The designation provides regulatory, financial
and commercial incentives to develop therapies for rare diseases
defined as having a prevalence of less than five in 10,000 people
in the European Union.
About NTLA-2001 NTLA-2001 is the first
experimental CRISPR therapy to be administered systemically, or
through a vein, to edit a gene inside the human body. Intellia’s
proprietary non-viral platform utilizes lipid nanoparticles
designed to deliver to the liver a simple, two-part genome
editing system: guide RNA specific to the disease-causing
gene and messenger RNA that encodes the Cas9 protein.
Intellia’s robust preclinical data showing deep and long-lasting
transthyretin (TTR) reduction following knockout of the target
gene in vivo support NTLA-2001’s potential as a single
dose of treatment. NTLA-2001 is part of a
co-development/co-promotion agreement between Intellia, the lead
development and commercialization party, and Regeneron
Pharmaceuticals, Inc.
About Transthyretin Amyloidosis (ATTR)
Transthyretin amyloidosis, or ATTR, is a rare, progressive and
fatal disease. Hereditary ATTR (hATTR) occurs when a person is born
with a specific DNA mutation in the TTR gene, which causes the
liver to produce a protein called transthyretin (TTR) in a
misfolded form and build up in the body. hATTR can manifest as
polyneuropathy (hATTR-PN), which can lead to nerve damage, or
cardiomyopathy (hATTR-CM), which involves heart muscle disease that
can lead to heart failure. In addition, non-mutated, or wild-type
TTR protein, can also accumulate in the body, leading to wild-type
ATTR (wtATTR). There are an estimated 50,000 hATTR patients
worldwide and between 200,000 and 500,000 people with wtATTR.
About Intellia Therapeutics Intellia
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 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: being able to complete clinical studies for
NTLA-2001 for the treatment of transthyretin amyloidosis (“ATTR”)
pursuant to its clinical trial applications (“CTA”), including
submitting additional regulatory applications in other countries;
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; 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, including but not
limited to its collaborations with Regeneron, including its
co-development programs for ATTR; and statements regarding the
timing of regulatory filings regarding its development
programs.
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 with Regeneron or its other
collaborations 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 (“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:Lina LiDirectorInvestor
Relations+1-857-706-1612 lina.li@intelliatx.com
Media:Julie FergusonInterim Head of External
Affairs & Communications+1-312-385-0098
julie.ferguson@intelliatx.com
Intellia Therapeutics (NASDAQ:NTLA)
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