Intellia Therapeutics to Present Updated Interim Clinical Data from Ongoing Phase 1/2 Study of NTLA-2002 for the Treatment of Hereditary Angioedema at the 2022 ACAAI Annual Scientific Meeting
31 Octobre 2022 - 12:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing potentially curative
therapeutics leveraging CRISPR-based technologies, today announced
that an abstract featuring new interim clinical data from the Phase
1/2 study of NTLA-2002 has been selected for a Distinguished
Industry Abstract oral presentation at the American College of
Allergy, Asthma & Immunology (ACAAI) 2022 Annual Scientific
Meeting, taking place November 10 – 14 in Louisville, Kentucky.
NTLA-2002 is an investigational in vivo CRISPR/Cas9 therapy in
development as a single-dose treatment to prevent angioedema
attacks in people living with hereditary angioedema (HAE). Building
on data released at the 2022 Bradykinin Symposium, the presentation
will include additional interim safety, kallikrein reduction and
attack rate data from the ongoing dose-escalation portion of
Intellia’s first-in-human study of NTLA-2002.
Presentation DetailsTitle: "In
vivo CRISPR/Cas9 editing of KLKB1 in Patients with Hereditary
Angioedema: A First-in-Human Study” Date and Time:
Saturday, November 12, 2022, from 4:30 – 5:30 p.m.
ETSession: Distinguished Industry Oral Abstracts -
Session APresenter: Dr. Hilary Longhurst, M.D.,
Ph.D., FRCP, FRCPath, Senior Medical Officer at Auckland District
Health Board and Honorary Associate Professor at University of
Auckland, New Zealand, the trial’s principal investigator in New
Zealand
Intellia Therapeutics Investor EventIntellia
will host a live webcast on Monday, November 14, 2022, at 8:00 a.m.
ET to review the presented data. To join the webcast, please visit
this link, or the Events and Presentations page of the Investors
& Media section of the company’s website at www.intelliatx.com.
A replay of the webcast will be available on Intellia’s website for
at least 30 days following the call.
About the NTLA-2002 Clinical ProgramIntellia’s
multi-national Phase 1/2 study is evaluating the safety,
tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in
adults with Type I or Type II hereditary angioedema (HAE). This
includes the measurement of plasma kallikrein protein levels and
activity as determined by HAE attack rate measures. The Phase 1
portion of the study is an open-label, single-ascending dose design
used to identify up to two dose levels of NTLA-2002 that will be
further evaluated in the randomized, placebo-controlled Phase 2
portion of the study. This Phase 1/2 study will identify the dose
of NTLA-2002 for use in future studies. Visit
clinicaltrials.gov (NCT05120830) for more details.
About NTLA-2002
Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2002
is the first single-dose investigational treatment being explored
in clinical trials for the potential to continuously reduce
kallikrein activity and prevent attacks in people living with
hereditary angioedema (HAE). NTLA-2002 is a wholly owned
investigational CRISPR therapeutic candidate designed to inactivate
the kallikrein B1 (KLKB1) gene, which encodes for
prekallikrein, the kallikrein precursor protein. NTLA-2002 is
Intellia’s second investigational CRISPR therapeutic candidate
to be administered systemically, by intravenous infusion, to edit
disease-causing genes inside the human body with a single dose of
treatment. 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
together carry out the precision editing.
About Hereditary Angioedema
Hereditary angioedema (HAE) is a rare, genetic disorder
characterized by severe, recurring and unpredictable inflammatory
attacks in various organs and tissues of the body, which can be
painful, debilitating and life-threatening. It is estimated that
one in 50,000 people are affected by HAE, and current treatment
options often include life-long therapies, which may require
chronic intravenous (IV) or subcutaneous (SC) administration as
often as twice per week, or daily oral administration to ensure
constant pathway suppression for disease control. Despite chronic
administration, breakthrough attacks still occur. Kallikrein
inhibition is a clinically validated strategy for the preventive
treatment of HAE attacks.
About Intellia TherapeuticsIntellia
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 Twitter @intelliatx.
Intellia Contacts:
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communications+1-857-449-4175ian.karp@intelliatx.com
Lina LiSenior Director, Investor Relations and Corporate
Communications +1-857-706-1612lina.li@intelliatx.com
Media:Rebecca SpaldingTen Bridge
Communications+1-646-509-3831media@intelliatx.com
rebecca@tenbridgecommunications.com
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