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 GTC3291
Trial ID NCT05667493
Disease Transthyretin Amyloid Cardiomyopathy
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
PhasePhase3
Recruitment statusRecruiting
TitleAn Open-Label Extension Study to Assess the Long-Term Safety of Eplontersen (ION-682884) in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
Year2022
CountryAustralia|Austria|Belgium|Canada|Germany|Greece|Israel|Italy|Portugal|Spain|Sweden|United States
Company sponsorIonis Pharmaceuticals, Inc.
Other ID(s)ION-682884-CS12|2022-000826-74
Vector information
VectorNo vector was used

Clinical Result

Cohort 1
Administration route subcutaneous injection
Dosage Eplontersen, once every month, up to 36 months
Pts 1400
Age Adult, Older_Adult

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