2021/06/26 by Julian D. Gillmore, Ed Gane, Jörg Täubel +23 · 14 citations
Biochemistry, Genetics and Molecular Biology · Medicine · #Amyloidosis: Diagnosis, Treatment, Outcomes #Lysosomal Storage Disorders Research #Sarcoidosis and Beryllium Toxicity Research
paper · pdf · doi:10.1056/nejmoa2107454
openalex publication_date 2021/06/26 · openalex created_date 2025/10/10 · openalex updated_date 2026/07/31
BACKGROUND: . METHODS: After conducting preclinical in vitro and in vivo studies, we evaluated the safety and pharmacodynamic effects of single escalating doses of NTLA-2001 in six patients with hereditary ATTR amyloidosis with polyneuropathy, three in each of the two initial dose groups (0.1 mg per kilogram and 0.3 mg per kilogram), within an ongoing phase 1 clinical study. RESULTS: after a single dose. Serial assessments of safety during the first 28 days after infusion in patients revealed few adverse events, and those that did occur were mild in grade. Dose-dependent pharmacodynamic effects were observed. At day 28, the mean reduction from baseline in serum TTR protein concentration was 52% (range, 47 to 56) in the group that received a dose of 0.1 mg per kilogram and was 87% (range, 80 to 96) in the group that received a dose of 0.3 mg per kilogram. CONCLUSIONS: . (Funded by Intellia Therapeutics and Regeneron Pharmaceuticals; ClinicalTrials.gov number, NCT04601051.).