MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)
Intellia Therapeutics is advancing NTLA-2001, a CRISPR-based therapeutic targeting hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). The Phase 3 MAGNITUDE-2 trial aims to establish the efficacy and safety of a single infusion of NTLA-2001 compared to placebo. Given the increasing prevalence of ATTRv-PN and the limited treatment options currently available, successful outcomes could position NTLA-2001 as a leading therapy in a niche but growing market. The collaboration with Regeneron Pharmaceuticals may enhance development capabilities and market reach. However, the competitive landscape includes other emerging therapies targeting TTR, necessitating close monitoring of their clinical progress and market strategies.
Indication: Neuromuscular Disease
Modality: protein therapy
Target: Transthyretin (TTR) protein silencing via CRISPR technology.
Sponsor: Intellia Therapeutics
Source URL: ClinicalTrials.gov
Source updated: Detailed source ingestion pending
Ingested: Jun 23, 2026
Model: trialsignal-ai-v1
Validation: validated
Matched by target_normalized: Transthyretin (TTR) protein silencing via CRISPR technology.
View original source fields
Condition raw: Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease, Neurodegenerative Disease, Hereditary, Neurodegenerative Diseases, Neuromuscular Diseases, Nerve Disorders, Nervous System Disease, Nervous System Diseases, Genetic Disease, Inborn, Amyloidosis, Familial, Amyloidosis, Hereditary, Amyloidosis, Polyneuropathies, Amyloid Neuropathies, Amyloid Neuropathies, Familial, Peripheral Nervous System Disease, Peripheral Nervous System Diseases, Metabolism, Inborn Errors, Metabolic Diseases
Condition normalized: Neuromuscular Disease, Neuromuscular Diseases (NMD), Neurodegenerative Disease, Neurodegenerative Disease, Hereditary, Neurodegenerative Diseases, Neuromuscular Diseases, Nerve Disorders, Nervous System Disease, Nervous System Diseases, Genetic Disease, Inborn, Amyloidosis, Familial, Amyloidosis, Hereditary, Amyloidosis, Polyneuropathies, Amyloid Neuropathies, Amyloid Neuropathies, Familial, Peripheral Nervous System Disease, Peripheral Nervous System Diseases, Metabolism, Inborn Errors, Metabolic Diseases
Modality raw: protein therapy
Modality normalized: protein therapy
Target raw: Transthyretin (TTR) protein silencing via CRISPR technology.
Target normalized: Transthyretin (TTR) protein silencing via CRISPR technology.