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RXI-147
- Development stage
- Preclinical
- Lead developer
- Phio 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
- Ophthalmic
01
Overview
RXI-147 is a self-delivering RNA interference (sd-rxRNA) therapeutic candidate designed to silence the expression of Vascular Endothelial Growth Factor (VEGF). Developed by Phio Pharmaceuticals (formerly RXi Pharmaceuticals), the sd-rxRNA technology features chemically modified, asymmetric siRNA molecules that incorporate a phosphorothioate backbone and hydrophobic modifications, allowing for spontaneous cellular uptake without the need for specialized delivery vehicles like lipid nanoparticles. By targeting VEGF mRNA for degradation via the RNA-induced silencing complex (RISC), RXI-147 inhibits the production of VEGF protein, a key driver of pathological neovascularization and vascular leakage. The drug is primarily investigated for the treatment of retinal diseases, including wet age-related macular degeneration (wet AMD) and diabetic retinopathy.
- Other names
- anti-vascular endothelial growth factor sd-rxRNAanti-VEGF sd-rxRNAself-delivering RNAi targeting VEGF
02
Targets
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