Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DJ5XY8
|
|||
| Drug Name |
SLN124
|
|||
| Drug Type |
Small interfering RNA
|
|||
| Indication | Beta thalassemia [ICD-11: 3A50.2; ICD-10: D56.1, D56.3] | Phase 1 | [1] | |
| Company |
Silence Therapeutics
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | TMPRSS6 messenger RNA (TMPRSS6 mRNA) | Target Info | Inhibitor | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04718844) A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome. U.S. National Institutes of Health. | |||
| REF 2 | SLN124, a GalNAc-siRNA Conjugate Targeting TMPRSS6, Efficiently Prevents Iron Overload in Hereditary Haemochromatosis Type 1. Hemasphere. 2019 Oct 1;3(6):e301. | |||
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