Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DJ0E7P
|
|||
| Drug Name |
ALN-TTR01
|
|||
| Drug Type |
Antisense oligonucleotide
|
|||
| Indication | Amyloidosis [ICD-11: 5D00; ICD-9: 277.3] | Phase 1 | [1] | |
| Transthyretin familial amyloid cardiomyopathy [ICD-11: 5D00.20; ICD-10: E85.1] | Phase 1 | [2] | ||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Transthyretin messenger RNA (TTR mRNA) | Target Info | Inhibitor | [3] |
| 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 | Clinical pipeline report, company report or official report of Alnylam Pharmaceuticals, Inc (2011). | |||
| REF 2 | ClinicalTrials.gov (NCT01148953) Trial to Evaluate Safety and Tolerability of ALN-TTR01 in Transthyretin (TTR) Amyloidosis. U.S. National Institutes of Health. | |||
| REF 3 | Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013 Aug 29;369(9):819-29. | |||
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