TRANSMEMBRANE PROTEASE, SERINE 6 (TMPRSS6) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
TWI939479BActive Publication Date: 2026-09-21ALNYLAM PHARMACEUTICALS INC
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Patent Information
- Application Number
- TW111115644
- Authority / Receiving Office
- TW · TW
- Patent Type
- Patents
- Current Assignee / Owner
- Priority Date
- 2021-11-11
- Filing Date
- 2022-04-25
- Publication Date
- 2026-09-21
- Estimated Expiration
- 2042-04-24
Abstract
This invention relates to RNAi agents, such as double-stranded RNA (dsRNA) agents, targeting the transmembrane protease (serine 6) (TMPRSS6) gene. This invention also relates to methods for using such RNAi agents to inhibit the expression of the TMPRSS6 gene and methods for preventing and treating TMPRSS6-related disorders, such as iron overload-related disorders and / or disorders of ineffective erythropoiesis, such as hereditary hemoglobinemia, β-thalassemia (e.g., severe and moderate β-thalassemia), polycythemia vera, myelometaplasia syndrome, congenital dysplasia, pyruvate kinase deficiency, erythropoietinosis, Parkinson's disease, Alzheimer's disease, and Friedrich's ataxia.
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