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Sirna-027
- Development stage
- Discontinued
- Lead developer
- Alnylam Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravitreal
01
Overview
Sirna-027 is a chemically modified short interfering RNA (siRNA) designed to target and silence the expression of vascular endothelial growth factor receptor 1 (VEGFR‑1) mRNA. By inhibiting VEGFR‑1, which is primarily found on vascular endothelial cells and stimulated by both VEGF and placental growth factor (PlGF), Sirna‑027 aims to reduce pathological angiogenesis associated with diseases such as age-related macular degeneration (AMD). The drug was developed by Sirna Therapeutics, later co-developed with Allergan, for the treatment of wet AMD. Clinical trials demonstrated biological activity and safety in early phases; however, development was halted after phase II due to insufficient efficacy compared to standard treatments[1][3][4][5][8].
- Other names
- SIRNA-027 (small interfering RNA directed against vascular endothelial growth factor receptor 1 mRNA)Agn-211745Agn211745Agn 211745
02
Targets
VEGFR-1 (Vascular endothelial growth factor receptor 1)
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