Drug intelligence / Profile preview

ASP2020

Development stage
Phase 1
Lead developer
Astellas Pharma
Modality
Stem Cell Therapies → Cell Therapies
Administration
Ophthalmic
01

Overview

ASP2020 is an investigational allogeneic cell therapy comprising human stem cell-derived retinal pigment epithelial (RPE) cells. Developed by the Astellas Institute for Regenerative Medicine (AIRM), it is designed for the treatment of Stargardt-type macular dystrophies, including Stargardt disease (STGD). The therapy is administered as a single subretinal injection, with the goal of replacing damaged or lost RPE cells in the macula. By replenishing the RPE layer, ASP2020 aims to restore the metabolic and structural support necessary for photoreceptor survival, potentially halting or reversing the progression of central vision loss in patients with these inherited retinal conditions.

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