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RTX-700

Development stage: Discontinued
Lead developer: River 3 Renal Corp
Modality: Tissue Cells → Other Cell Types → Cell Therapies
Administration: Intravenous

Overview

RTX-700 is an engineered red blood cell therapy developed by Rubius Therapeutics for the treatment of Pompe disease (glycogen storage disease type II). Utilizing the company's Red Cell Medicine platform, RTX-700 consists of allogeneic red blood cells engineered to express human acid alpha-glucosidase (GAA). The therapy was designed to provide a sustained source of the enzyme to degrade accumulated glycogen in tissues while potentially reducing the immunogenicity and frequent dosing requirements associated with traditional enzyme replacement therapies. Development of RTX-700 was discontinued following Rubius Therapeutics' strategic pivot to oncology in 2020 and the company's subsequent liquidation in 2023.

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