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cemdisiran
- Development stage
- Phase 3
- Lead developer
- Regeneron Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Peptide-Drug Conjugates → Peptide Conjugates → Peptides, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Cemdisiran is a chemically modified small interfering RNA (siRNA) therapeutic conjugated to N-acetylgalactosamine (GalNAc) for targeted delivery to hepatocytes. It is designed to silence the gene encoding complement component 5 (C5), thereby reducing C5 protein production in the liver and inhibiting activation of the terminal complement pathway. This mechanism addresses diseases driven by overactivation of the complement system, such as paroxysmal nocturnal hemoglobinuria (PNH), myasthenia gravis, dry macular degeneration, haemolytic uraemic syndrome, and immunoglobulin A nephropathy. Cemdisiran is being developed by Alnylam Pharmaceuticals and Regeneron Pharmaceuticals and has been evaluated both as monotherapy and in combination with other agents like pozelimab[1][3][6][7].
02
Targets
C5
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