Intellia Therapeutics to Present Longer-Term Data from the Ongoing Phase 1 Clinical Trial of Nexiguran Ziclumeran (nex-z) for the Treatment of Hereditary Transthyretin (ATTR) Amyloidosis with Polyneuropathy

Core Insights - Intellia Therapeutics is set to present long-term data from its Phase 1 trial of investigational nex-z for hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) at the 5th International ATTR Amyloidosis Meeting on September 25-26, 2025, in Baveno, Italy, highlighting up to three years of patient follow-up [1] Company Overview - Intellia Therapeutics, Inc. is a clinical-stage gene editing company focused on CRISPR-based therapies, aiming to revolutionize medicine by developing novel treatments for unmet medical needs [5] - The company is leading the development and commercialization of nex-z in collaboration with Regeneron Pharmaceuticals, Inc. [3] Product Information - Nex-z is based on CRISPR/Cas9 technology and aims to be the first one-time treatment for transthyretin (ATTR) amyloidosis by inactivating the TTR gene responsible for producing the TTR protein [3] - Interim Phase 1 clinical data indicates that nex-z administration results in consistent, deep, and long-lasting reductions in TTR levels [3] Disease Context - ATTR amyloidosis is a rare and progressive disease, with hereditary ATTR (ATTRv) caused by mutations in the TTR gene, leading to the production of misfolded TTR proteins that accumulate as amyloid in the body [4] - Approximately 50,000 individuals globally are affected by ATTRv amyloidosis, while the number of those with wild-type ATTR (ATTRwt) amyloidosis ranges between 200,000 and 500,000 [4]