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ART-020

Development stage: Preclinical
Lead developer: ART BioScience
Modality: Gene Editing → Gene Therapies, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intravenous

Overview

ART-020 is a preclinical gene-editing therapeutic candidate developed by ART BioScience (also known as ART-mRNA) for the treatment of Duchenne Muscular Dystrophy (DMD). The program utilizes a gene-editing mechanism, likely delivered via the company's proprietary mRNA platform, to address mutations in the dystrophin gene. By correcting or bypassing genetic defects, the therapy aims to restore the production of functional dystrophin protein in muscle cells. As of 2024, ART-020 is in the discovery and early optimization stages of development.

Targets

DMD (Dystrophin)

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