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GTB-USH-CRISPR

Development stage: Preclinical
Lead developer: GeneToBe
Modality: CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies

Overview

GTB-USH-CRISPR is an experimental CRISPR-based gene editing therapy being developed by GeneToBe for the treatment of Usher syndrome. The program utilizes CRISPR technology to correct or modify disease-causing genetic variants that lead to the combined hearing and vision loss characteristic of the condition. As of early 2024, the program is in the late discovery stage of development. Specific molecular targets within the Usher syndrome gene complex have not been publicly disclosed by the developer.

Other names: Usher syndrome program

Targets

USH2A (Usherin)

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