Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DW95VT
|
|||
| Drug Name |
NTLA-2001
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Transthyretin amyloidosis [ICD-11: 5D00] | Phase 1 | [1] | |
| Company |
Intellia Therapeutics Cambridge, MA Regeneron Tarrytown, NY
|
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Transthyretin (TTR) | Target Info | . | [2] |
| Pathway Interaction Database | FOXA2 and FOXA3 transcription factor networks | |||
| Reactome | Retinoid cycle disease events | |||
| The canonical retinoid cycle in rods (twilight vision) | ||||
| Non-integrin membrane-ECM interactions | ||||
| Retinoid metabolism and transport | ||||
| Amyloid formation | ||||
| WikiPathways | Visual phototransduction | |||
| Extracellular matrix organization | ||||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04601051) 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). U.S.National Institutes of Health. | |||
| REF 2 | CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis. N Engl J Med. 2021 Aug 5;385(6):493-502. | |||
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