ADSSL1 CRISPR gene therapy

ADSSL1 CRISPR gene therapy is an investigational CRISPR-based gene editing treatment developed by Cure Rare Disease for the treatment of ADSSL1-related myopathy. The therapy utilizes CRISPR technology to target and correct pathogenic variants within the ADSSL1 gene, which encodes the muscle-specific enzyme adenylosuccinate synthetase like 1. This enzyme plays a crucial role in the purine nucleotide cycle, and its deficiency leads to progressive muscle weakness and wasting. By modifying the underlying genetic defect, the treatment aims to restore functional enzyme levels and improve clinical outcomes for patients with this rare distal myopathy. It is currently in preclinical development (Stage 1-2).

Other names

ADSSL1 gene editing therapyADSSL-1 gene editing therapyADSSL 1 gene editing therapyCRISPR-ADSSL1CRISPR-ADSSL-1CRISPR-ADSSL 1