| 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 | GTC0671 |
| Trial ID | 2019-001698-10 |
| Disease | Familial Amyloid Polyneuropathy |
| Altered gene | TTR |
| Therapeutic/Target gene | Target gene |
| Therapy | ASO |
| Treatment | AKCEA-TTR-LRx|Eplontersen|ION-682884|IONIS-TTR-LRx|ION-682884|Wainua |
| Location approved | US |
| Phase | Phase3 |
| Recruitment status | Active, Not Recruiting |
| Title | A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy |
| Year | 2020 |
| Country | Argentina|Australia|Austria|Belgium|Brazil|Canada|Czechia|Denmark|France|Germany|Greece|Israel|Italy|Japan|Poland|Portugal|Puerto Rico|Spain|Sweden|United Kingdom|United States |
| Company sponsor | Ionis Pharmaceuticals, Inc. |
| Other ID(s) | NCT04136171 |
| Vector information | |||
|
|||
| Cohort 1 | |||||||
|
|||||||