Regorafenib Nanoparticles for the Treatment of Age-Related Macular Degeneration

Authors

• Megan Gearinger, St. John Fisher University
• Lisa Daitz, St. John Fisher University
• Priyanka Bhatt, St. John Fisher UniversityFollow

Document Type

Poster Presentation

Publication Date

4-17-2026

Keywords

fsc2026

Abstract

Background: Age-related macular degeneration (AMD) is a leading cause of blindness, with advanced neovascular AMD (nAMD) driven by choroidal neovascularization. Regorafenib (REG) is a potent multi-kinase inhibitor with broad anti-angiogenic activity; however, its ocular application is severely limited by systemic toxicity and poor bioavailability. Encapsulating REG within polymeric nanoparticles offers a promising strategy to enhance localized ocular delivery while minimizing toxicity.

Objective: To evaluate the cytotoxicity, anti-angiogenic activity, and cellular uptake of regorafenib-loaded mPEG-PLGA nanoparticles (REG NPs) in human retinal pigment epithelial (ARPE-19) cells as a potential therapeutic platform for nAMD.

Methods: Cytotoxicity of REG NPs, free REG solution, and placebo NPs was evaluated using an MTT assay in ARPE-19 cells over 24 and 48 hours. Anti-angiogenic efficacy was quantified via anti-VEGF ELISA and evaluated through wound scratch assays. Cellular uptake of coumarin-6-loaded NPs was visualized and analyzed using confocal microscopy.

Results: REG NPs maintained an acceptable cytotoxicity profile, indicating that nanoparticle encapsulation effectively mitigates the known toxicity of free regorafenib. Furthermore, REG NPs demonstrated stronger anti-angiogenic effects than the free drug solution across multiple in vitro assays. The nanoparticle formulation significantly reduced VEGF secretion for extended durations and limited cell migration. Confocal microscopy confirmed enhanced intracellular uptake of the NPs, which supports their increased potency at equivalent coumarin-6 concentrations.

Conclusion: REG NPs exhibited a highly favorable in vitro safety and efficacy profile in ARPE-19 cells. These findings strongly justify advancing this formulation into complex, hypoxia-driven in vivo models to evaluate its therapeutic potential for nAMD.

Comments

Poster presented at the 2026 Fisher Showcase, St. John Fisher University, April 17, 2026.

Publication Information

Gearinger, Megan; Daitz, Lisa; and Bhatt, Priyanka, "Regorafenib Nanoparticles for the Treatment of Age-Related Macular Degeneration" (2026). Fisher Showcase 2026. Paper 38.
https://fisherpub.sjf.edu/fsc2026/38

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