Target Information
| Target General Information | Top | |||||
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| Target ID |
T23389
(Former ID: TTDC00329)
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| Target Name |
Transthyretin messenger RNA (TTR mRNA)
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| Synonyms |
Transthyretin (mRNA); TBPA (mRNA); Prealbumin (mRNA); PALB (mRNA); ATTR (mRNA)
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| Gene Name |
TTR
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| Target Type |
Successful target
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[1] | ||||
| Disease | [+] 1 Target-related Diseases | + | ||||
| 1 | Amyloidosis [ICD-11: 5D00] | |||||
| Function |
Probably transports thyroxine from the bloodstream to the brain. Thyroid hormone-binding protein.
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| BioChemical Class |
mRNA target
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| UniProt ID | ||||||
| Sequence |
MASHRLLLLCLAGLVFVSEAGPTGTGESKCPLMVKVLDAVRGSPAINVAVHVFRKAADDT
WEPFASGKTSESGELHGLTTEEEFVEGIYKVEIDTKSYWKALGISPFHEHAEVVFTANDS GPRRYTIAALLSPYSYSTTAVVTNPKE Click to Show/Hide
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| Drugs and Modes of Action | Top | |||||
|---|---|---|---|---|---|---|
| Approved Drug(s) | [+] 3 Approved Drugs | + | ||||
| 1 | Eplontersen | Drug Info | Approved | Amyloid polyneuropathy | [2] | |
| 2 | Patisiran | Drug Info | Approved | Hereditary amyloidosis | [3] | |
| 3 | Vutrisiran | Drug Info | Approved | Hereditary amyloidosis | [4] | |
| Clinical Trial Drug(s) | [+] 3 Clinical Trial Drugs | + | ||||
| 1 | ISIS-TTR | Drug Info | Phase 3 | Amyloidosis | [5] | |
| 2 | Revusiran | Drug Info | Phase 3 | Amyloidosis | [6] | |
| 3 | ALN-TTR01 | Drug Info | Phase 1 | Amyloidosis | [7] | |
| Mode of Action | [+] 1 Modes of Action | + | ||||
| Inhibitor | [+] 2 Inhibitor drugs | + | ||||
| 1 | Vutrisiran | Drug Info | [8] | |||
| 2 | ALN-TTR01 | Drug Info | [10] | |||
| Cell-based Target Expression Variations | Top | |||||
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| Cell-based Target Expression Variations | ||||||
| Target Affiliated Biological Pathways | Top | |||||
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| PID Pathway | [+] 1 PID Pathways | + | ||||
| 1 | FOXA2 and FOXA3 transcription factor networks | |||||
| Reactome | [+] 5 Reactome Pathways | + | ||||
| 1 | Retinoid cycle disease events | |||||
| 2 | The canonical retinoid cycle in rods (twilight vision) | |||||
| 3 | Non-integrin membrane-ECM interactions | |||||
| 4 | Retinoid metabolism and transport | |||||
| 5 | Amyloid formation | |||||
| WikiPathways | [+] 2 WikiPathways | + | ||||
| 1 | Visual phototransduction | |||||
| 2 | Extracellular matrix organization | |||||
| References | Top | |||||
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| REF 1 | Clinical pipeline report, company report or official report of Alnylam Pharmaceuticals, Inc. | |||||
| REF 2 | FDA Approved Drug Products from FDA Official Website. 2023. Application Number: 217388 | |||||
| REF 3 | 2018 FDA drug approvals.Nat Rev Drug Discov. 2019 Feb;18(2):85-89. | |||||
| REF 4 | FDA Approved Drug Products from FDA Official Website. 2023. Application Number: 215515. | |||||
| REF 5 | ClinicalTrials.gov (NCT02175004) Open-Label Extension Assessing Long Term Safety and Efficacy of ISIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP). U.S. National Institutes of Health. | |||||
| REF 6 | ClinicalTrials.gov (NCT02319005) ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC). U.S. National Institutes of Health. | |||||
| REF 7 | Clinical pipeline report, company report or official report of Alnylam Pharmaceuticals, Inc (2011). | |||||
| REF 8 | Clinical pipeline report, company report or official report of Alnylam Pharmaceuticals. | |||||
| REF 9 | Clinical pipeline report, company report or official report of ISIS Pharmaceuticals (2011). | |||||
| REF 10 | Safety and efficacy of RNAi therapy for transthyretin amyloidosis. N Engl J Med. 2013 Aug 29;369(9):819-29. | |||||
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