| 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 | GTC0695 |
| Trial ID | 2021-001427-40 |
| 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 | Ongoing |
| Title | An Open-Label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy |
| Year | 2022 |
| Country | Portugal |
| Company sponsor | Ionis Pharmaceuticals, Inc. |
| Vector information | |||
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| Cohort 1 | |||||||
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