Data presented in late-breaking oral presentation demonstrated deep and consistent TTR reduction following a single dose of NTLA-2001 in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM) Greater than 90% mean serum TTR reductions after a single dose of NTLA-2001 were […]
Tag: Intellia
Intellia and Regeneron Announce Initial Data from the Cardiomyopathy Arm of Ongoing Phase 1 Study of NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis
Interim data from the cardiomyopathy arm of the Phase 1 study of NTLA-2001 showed deep and sustained mean serum transthyretin (TTR) reductions of 93% and 92% at 0.7 mg/kg and 1.0 mg/kg doses, respectively, at day 28 NTLA-2001 was generally […]