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 GTC3149
Trial ID NCT05505838
Disease Transthyretin Amyloid Cardiomyopathy
Altered gene TTR
Therapeutic/Target gene Target gene
TherapysiRNA
Treatment ALN-TTR02|ONPATTRO|patisiran
Location approved US, EU, UK, Japan, Canada, Switzerland, Brazil, China, Israel, Turkey, Australia
Recruitment statusNo Longer Available
TitleExpanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis (ATTR Amyloidosis) With Cardiomyopathy
Company sponsorAlnylam Pharmaceuticals
Other ID(s)ALN-TTR02-014
Vector information
Vectorlipid nanoparticle

Clinical Result

Cohort 1
Administration route None
Age Adult, Older_Adult

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