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Gap Junction Therapeutics genome editing therapy
- Development stage
- Preclinical
- Lead developer
- Gap Junction
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Local Injection
01
Overview
Gap Junction Therapeutics genome editing therapy is an experimental gene-based treatment designed to address hereditary hearing loss. Developed by Gap Junction Therapeutics, a spin-off from Juntendo University, the therapy utilizes genome editing technology to correct mutations in gap junction molecules, specifically connexin proteins, within the inner ear cells. By restoring the functional integrity of these gap junctions, the therapy aims to recover hearing in patients with genetic defects. As of early 2025, the program is in the preclinical stage, having demonstrated success in mouse models.
- Other names
- connexin gene therapygap junction gene therapy
02
Targets
GJB2 (Gap junction protein beta-2)
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