| Gene symbol | TTR | Synonyms | ATTR, CTS, CTS1, HEL111, HsT2651, PALB, TBPA, TTN | Type of gene | protein-coding |
| Chromosome | 18 | Map location | 18q12.1 | dbXrefs | |
| Gene perturbation-related omics dataset | PerturbAtlas | ||||
| Description | transthyretin | ||||
| GTO ID | GTC1471 |
| Trial ID | NCT02053454 |
| Disease | Amyloidosis |
| Altered gene | TTR |
| Therapeutic/Target gene | Target gene |
| Therapy | siRNA |
| Treatment | ALN-TTR02|ONPATTRO|patisiran |
| Location approved | US, EU, UK, Japan, Canada, Switzerland, Brazil, China, Israel, Turkey, Australia |
| Phase | Phase1 |
| Recruitment status | Completed |
| Title | A Phase 1, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-TTR02 in Japanese Healthy Volunteers |
| Year | 2014 |
| Country | United Kingdom |
| Company sponsor | Alnylam Pharmaceuticals |
| Other ID(s) | ALN-TTR02-005 |
| Vector information | |||
|
|||
| Cohort1: patisiran | |||||||
|
|||||||
| Cohort2: Sterile Normal Saline | |||||||
|
|||||||