参考链接: 1.https://ir.intelliatx.com/news-releases/news-release-details/intellia-and-regeneron-present-updated-interim-data-phase-1 2. https://www.nejm.org/doi/full/10.1056/NEJMoa2107454
参考资料: [1] Intellia and Regeneron Announce Updated Phase 1 Data Demonstrating a Single Dose of NTLA-2001, Investigational CRISPR Therapy for Transthyretin (ATTR) Amyloidosis, Resulted in Rapid, Deep and Sustained Reduction in Disease-Causing Protein, Retrieved February 28, 2022, from https://www...
Intellia’s global Phase 1 trial is an open-label, multi-center, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). The trial’s primary objectives are to assess the ...
2001 via intravenous administration. The study will enroll up to 38 participants (ages 18-80 years) and consists of a single-ascending dose phase in Part 1 and, following the identification of an optimal dose, an expansion cohort in Part 2. Visitclinicaltrials.gov(NCT04601051...