Drug Information
Drug General Information | Top | |||
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Drug ID |
DHTN76
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Drug Name |
ALN-CC5
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Drug Type |
Antisense oligonucleotide
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Indication | Haemolytic uraemic syndrome [ICD-11: 3A21.2; ICD-10: D59.3] | Phase 2 | [1] | |
Company |
Alnylam Pharmaceuticals: Cemdisiran
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Target and Pathway | Top | |||
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Target(s) | C5 messenger RNA (C5 mRNA) | Target Info | Inhibitor | [2] |
References | Top | |||
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REF 1 | ClinicalTrials.gov (NCT03303313) A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome. U.S. National Institutes of Health. | |||
REF 2 | Investigational RNAi Therapeutic Targeting C5 Is Efficacious in Pre-clinical Models of Myasthenia Gravis. Mol Ther Methods Clin Dev. 2019 May 10;13:484-492. |
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