Intellia Therapeutics to Present Updated Data from Ongoing Phase 1 Study of NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis and Hold Conference Call to Discuss Third Quarter 2023 Earnings in November
26 Ottobre 2023 - 1:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing potentially curative
therapeutics leveraging CRISPR-based technologies, today announced
two upcoming events in November.
NTLA-2001 Interim Clinical Data Update Intellia
will present updated data from the ongoing NTLA-2001 Phase 1 study
for the treatment of transthyretin (ATTR) amyloidosis being
presented at the 4th International ATTR Amyloidosis Meeting, taking
place November 2 – 3 in Madrid, Spain.
- Presentation Details:
- Title: "Enabling the Development of Serum TTR
as a Biomarker for Treatment of ATTR Amyloidosis”
- Date and Time: Thursday, November 2, 2023,
from 11:30 a.m. – 1:00 p.m. CET
- Session: Latest Data for Patients’ Diagnosis
in a Changing World
- Presenter: Dr. Julian Gillmore, M.D, Ph.D.,
FRCP, FRCPath, Professor of Medicine, National Amyloidosis
Centre, UCL Division of Medicine, Royal Free
Hospital, U.K., the trial’s U.K. national coordinating
investigator
Third Quarter 2023 Earnings Intellia will host
a webcast to discuss its third quarter 2023 financial results and
operational highlights on November 9, 2023, at 8 a.m. ET. The
Company will also review the NTLA-2001 data presented at the 4th
International ATTR Amyloidosis Meeting.
- To join the webcast, please visit this link, or the Events and
Presentations page of the Investors & Media section on
Intellia’s website at www.intelliatx.com.
- A replay of the call will be available beginning on November 9,
2023, at 12 p.m. ET.
About NTLA-2001Based on Nobel
Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially
be the first single-dose treatment for ATTR amyloidosis. NTLA-2001
is the first investigational CRISPR therapy candidate to be
administered systemically, or through a vein, to edit genes inside
the human body. Intellia’s proprietary non-viral platform deploys
lipid nanoparticles to deliver to the liver a two-part genome
editing system: guide RNA specific to the disease-causing
gene and messenger RNA that encodes the Cas9 enzyme, which
carries out the precision editing. Robust preclinical and clinical
data, showing deep and long-lasting transthyretin (TTR) reduction
following in vivo inactivation of the target gene,
supports NTLA-2001’s potential as a single-administration
therapeutic. Intellia leads development and commercialization of
NTLA-2001 as part of a multi-target discovery, development and
commercialization collaboration with Regeneron. The
global Phase 1 trial is an open-label, multi-center, two-part study
of NTLA-2001 in adults with hereditary transthyretin amyloidosis
with polyneuropathy (ATTRv-PN) or transthyretin amyloidosis with
cardiomyopathy (ATTR-CM). The trial is now closed for enrollment.
Visit clinicaltrials.gov (NCT04601051) for more
details.
About Transthyretin (ATTR) Amyloidosis
Transthyretin amyloidosis, or ATTR amyloidosis, is a rare,
progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis
occurs when a person is born with mutations in
the TTR gene, which causes the liver to produce
structurally abnormal transthyretin (TTR) protein with a propensity
to misfold. These damaged proteins build up as amyloid in the body,
causing serious complications in multiple tissues, including the
heart, nerves and digestive system. ATTRv amyloidosis predominantly
manifests as polyneuropathy (ATTRv-PN), which can lead to nerve
damage, or cardiomyopathy (ATTRv-CM), which can lead to heart
failure. Some individuals without the genetic mutation produce
non-mutated, or wild-type TTR proteins that become unstable over
time, misfolding and aggregating in disease-causing amyloid
deposits. This condition, called wild-type ATTR (ATTRwt)
amyloidosis, primarily affects the heart. There are an estimated
50,000 people worldwide living with ATTRv amyloidosis and between
200,000 and 500,000 people with ATTRwt amyloidosis.
About Intellia Therapeutics
Intellia Therapeutics, a leading clinical-stage genome editing
company, is developing novel, potentially curative therapeutics
leveraging CRISPR-based technologies. To fully realize the
transformative potential of CRISPR-based technologies, Intellia is
pursuing two primary approaches. The company’s in vivo programs use
intravenously administered CRISPR as the therapy, in which
proprietary delivery technology enables highly precise editing of
disease-causing genes directly within specific target tissues.
Intellia’s ex vivo programs use CRISPR to create the therapy by
using engineered human cells to treat cancer and autoimmune
diseases. Intellia’s deep scientific, technical and clinical
development experience, along with its robust intellectual property
portfolio, have enabled the company to take a leadership role in
harnessing the full potential of genome editing to create new
classes of genetic medicine. Learn more at intelliatx.com. Follow
us on X (formerly known as Twitter) @intelliatx.
Intellia Contacts:
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communicationsian.karp@intelliatx.com
Lina LiSenior Director, Investor Relations and Corporate
Communicationslina.li@intelliatx.com
Media:Matt CrensonTen Bridge
Communicationsmedia@intelliatx.commcrenson@tenbridgecommunications.com
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