| 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 | GTC2486 |
| Trial ID | NCT04601051 |
| Disease | Transthyretin Amyloid Cardiomyopathy |
| Altered gene | TTR |
| Therapeutic/Target gene | Target gene |
| Therapy | Gene editing |
| Treatment | NTLA-2001 |
| Phase | Phase1 |
| Recruitment status | Active, Not Recruiting |
| Title | Phase 1 Two-Part (Open-label, Single Ascending Dose (Part 1) and Open-label, Single Dose Expansion (Part 2)) Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM) |
| Year | 2020 |
| Country | France|New Zealand|Sweden|United Kingdom |
| Company sponsor | Intellia Therapeutics |
| Other ID(s) | ITL-2001-CL-001|2020-002034-32 |
| Vector information | |||
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| Cohort1: Polyneuropathy | |||||||
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| Cohort2: Cardiomyopathy(UK only) | |||||||
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