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NTLA
Intellia Therapeutics, Inc.
Intellia Therapeutics develops CRISPR-based gene editing treatments for genetic and acquired diseases. The pipeline includes lonvoguran ziclumeran (lonvo-z) for hereditary angioedema, currently in Phase 3 trials with a potential FDA application filing targeted for late 2026, and nexiguran ziclumeran (nex-z) for transthyretin amyloidosis, which is working through an FDA clinical hold on its Phase 3 program. Both programs aim to deliver durable or potentially lifelong disease control through one-time treatments.
$13.48+51.97%1Y
NTLA · daily close · illustrative · 0 catalysts marked
Pipeline
Phase 3Hereditary Angioedema
Phase 3Neuromuscular Disease
Phase 3Neurodegenerative Disease
Phase 3Nerve Disorders
Phase 3Nervous System Disease
Phase 3Genetic Disease, Inborn
Phase 3Amyloidosis, Familial
Phase 3Amyloidosis, Hereditary
Phase 3Amyloidosis
Phase 3Polyneuropathies
Phase 3Amyloid Neuropathies
Phase 3Peripheral Nervous System Disease
Phase 3Metabolism, Inborn Errors
Phase 3Metabolic Diseases
NTLA-2001 (Intellia/Regeneron): in vivo lipid nanoparticle CRISPR-Cas9 therapy that knocks out TTR in hepatocytes. Phase 3 in transthyretin amyloidosis (ATTR-CM and hereditary ATTR-PN).
Phase 3Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Phase 3Hereditary Angioedema
Phase 2Hereditary Angioedema
phase3Hereditary Angioedema
Catalyst Calendar
JUN
Readoutlonvoguran ziclumeran
“The Company expects to report topline data from the HAELO Phase 3 trial by mid-2026.”
H2
NDAlonvoguran ziclumeran
“Intellia is preparing to complete the BLA filing in the second half of 2026”
H2
NDANTLA-2002
“its interactions with regulatory authorities, including the potential submission of a biologics license application for NTLA-2002 for the treatment of HAE in the second half of 2026”
H1
Conf.lonvoguran ziclumeran
“for a potential U.S. launch of lonvo-z in the first half of 2027, if approved”
Past (4)
2025
Readoutnexiguran ziclumeran
“Present longer-term data from both ATTR-CM and ATTRv-PN patients in the Phase 1 study – data will include updated measures of clinical efficacy and safety.”
2025
ReadoutNTLA-2002
“Present longer-term data from the Phase 1/2 study – data will include patients in the Phase 2 portion who initially received a 25 mg dose or placebo and were subsequently given the 50 mg dose of NTLA-2002 selected for the Phase 3 study.”
JAN 27
INDnexiguran ziclumeran
“On January 27, 2026, Intellia announced that the U.S. Food and Drug Administration ("FDA") has removed the clinical hold on the investigational new drug application ("IND") for the MAGNITUDE-2 Phase 3 clinical trial of nexiguran ziclumeran ("nex-z") for patients with hereditary transthyretin ("ATTR") amyloidosis with polyneuropathy ("ATTRv-PN").”