Basic Information

Gene symbol TTR Synonyms ATTR, CTS, CTS1, HEL111, HsT2651, PALB, TBPA, TTN Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas
Description transthyretin

GTO ID GTC2656
Trial ID NCT04843020
Disease Transthyretin Amyloidosis
Altered gene TTR
Therapeutic/Target gene Target gene
TherapyASO
Treatment AKCEA-TTR-LRx|Eplontersen|ION-682884|IONIS-TTR-LRx|ION-682884|Wainua
Location approved US
PhasePhase2
Recruitment statusNot Recruiting
TitleAn Open-Label Study of ION-682884 in Patients With TTR Amyloidosis Who Have Completed a 24-Open Label Study of Inotersen for TTR Amyloidosis Cardiomyopathy
Year2021
CountryUnited States
Company sponsorBrigham and Women's Hospital
Other ID(s)2021P000784
Vector information
VectorNo vector was used

Clinical Result

Cohort 1
Administration route subcutaneous injection
Dosage 45 mg, monthly
Age Child, Adult

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