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 GTC1572
Trial ID NCT02627820
Disease Senile Systemic Amyloidosis
Altered gene TTR
Therapeutic/Target gene Target gene
TherapyASO
Treatment ISIS 416858|IONIS-FXIRX|FXI-ASO|ISIS-FXI Rx|BAY2306001||BAY2306001|Fesomersen
PhasePhase2
Recruitment statusWithdrawn
TitleAn 18 Month Open Label Study Of The Tolerability And Efficacy Of An Antisense Oligonucleotide In Patients With Wild-Type Transthyretin Amyloid Cardiomyopathy (Senile Systemic Amyloidosis)
Year2015
CountryUnited States
Company sponsorBrigham and Women's Hospital
Other ID(s)2015-P001574
Vector information
VectorNo vector was used

Clinical Result

Cohort 1
Administration route subcutaneous injection
Age Adult, Older_Adult

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