Drug intelligence / Profile preview

OLX301D

Development stage
Preclinical
Lead developer
Olix 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, 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
Intravitreal
01

Overview

**OLX301D** is an investigational RNAi therapeutic developed by OliX Pharmaceuticals using proprietary asymmetric siRNA (asiRNA) technology to silence connective tissue growth factor (CTGF) mRNA. It targets subretinal fibrosis and neovascularization in wet age-related macular degeneration (wet AMD), addressing unmet needs in choroidal neovascularization and disciform scarring that cause vision loss via macular edema and retinal detachment. Preclinical studies in animal models demonstrated efficacy in inhibiting fibrosis progression and showed comparable effects to existing anti-VEGF therapies, positioning it as a potential first-in-class treatment for combination or non-responsive patients.[1][3][7][9]

02

Targets

CTGF (Connective tissue growth factor)

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