Drug intelligence / Profile preview

umedaptanib pegol

Development stage
Phase 2
Lead developer
Ribomic
Modality
RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravitreal, Subcutaneous
01

Overview

RBM-007, now known as umedaptanib pegol, is a novel oligonucleotide-based aptamer developed by Ribomic. It is composed of 37 nucleotides with ribose 2′ modifications for nuclease resistance and PEGylation to improve pharmacokinetics[3][6]. The drug specifically binds to and inhibits fibroblast growth factor 2 (FGF2), blocking its interaction with FGF receptors FGFR1–4[3]. This inhibition reduces both VEGF expression and FGF-driven neovascularization and fibrosis, providing dual anti-angiogenic and anti-scarring effects[1][5]. Umedaptanib pegol is being developed primarily for wet age-related macular degeneration (wet AMD) but has also been investigated in achondroplasia, cancer pain, lung cancer, and fibrosis[3][8]. Its mechanism of action distinguishes it from standard anti-VEGF therapies by targeting both angiogenesis and fibrotic pathways in disease processes[1][5].

02

Targets

FGF2

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