Intellia Initiates Phase 3 Study of Nex-z Gene Therapy for Hereditary ATTR Polyneuropathy
Intellia Therapeutics (NASDAQ:NTLA) has announced the first patient has been dosed in MAGNITUDE-2, a pivotal, global Phase 3 trial evaluating its one-time gene therapy, nexiguran ziclumeran (nex-z), for hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN).
Intellia’s President and CEO, Dr. John Leonard, expressed optimism that the study will demonstrate nex-z’s potential to halt or reverse disease progression with a single dose. The Phase 3 trial builds on Phase 1 data showing rapid and near-complete reduction of the disease-causing TTR protein.
Dr. Paulo Sgobbi of PSEG Clinical Research Center highlighted nex-z’s potential to redefine the treatment paradigm for this debilitating, progressive disease. Intellia anticipates sharing more Phase 1 data later this year and plans to file for FDA approval of nex-z for ATTRv-PN by 2028, pending positive Phase 3 results. The MAGNITUDE-2 trial is a randomized, double-blind, placebo-controlled study assessing the efficacy and safety of a single 55 mg infusion of nex-z in approximately 50 ATTRv-PN patients. The primary endpoints include changes in TTR levels and neuropathy impairment scores.
Nex-z is an investigational CRISPR-based therapy designed to inactivate the TTR gene in the liver, reducing the abnormal protein buildup that causes ATTR amyloidosis. It has received Orphan Drug and Regenerative Medicine Advanced Therapy designations. ATTR amyloidosis is a rare, progressive, and often fatal disease caused by misfolded TTR protein deposits. Current treatments are limited to slowing protein accumulation. Intellia is also developing nex-z for ATTR amyloidosis with cardiomyopathy. NTLA-2001 consists of a proprietary lipid nanoparticle (LNP) delivery system with liver tropism, carrying a single guide RNA (sgRNA) that targets human TTR and a human-codon–optimized mRNA sequence of Streptococcus pyogenes Cas9 protein.
https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-first-patient-dosed-magnitude-2
About PackGene
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
