Complement component irna compositions and methods of use thereof
iRNA compositions target CFB, C3, and C9 genes to inhibit complement activity, addressing the limitations of existing therapies for complement-related diseases like PNH and aHUS, providing a cost-effective and less invasive treatment.
JP2024170563APending Publication Date: 2024-12-10ALNYLAM PHARMACEUTICALS INC
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Patent Information
- Application Number
- JP2024153584
- Authority / Receiving Office
- JP · JP
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2013-12-12
- Filing Date
- 2024-09-06
- Publication Date
- 2024-12-10
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Figure 2024170563000001_ABST
Abstract
To provide: iRNA compositions which effect the RNA-induced silencing complex (RISC)-mediated cleavage of RNA transcripts of a complement factor B (CFB) gene; and methods of use thereof.SOLUTION: The invention relates to: iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement factor B (CFB) gene, the complement component C3 gene and the complement component C9 gene; and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of CFB, C9 and / or C3 and to treat subjects having a complement component-associated disease, e.g., paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndromes.SELECTED DRAWING: Figure 1
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