Gene editing system for the treatment of autosomal dominant disease-related genes

This technology is a modified CRISPR/Cas9 gene editing therapy that is mutation-independent for treating autosomal dominant disease-related genes, including retinitis pigmentosa.

Unmet Need: Simple treatment for autosomal dominant rhodopsin diseases

There are currently no known cures for autosomal dominant rhodopsin diseases including retinitis pigmentosa. Management of the disease is limited to vitamin supplements, protection from sunlight, and visual aids. Due to the diversity of underlying mutations in retinitis pigmentosa, traditional patient-specific therapy targeting specific mutations is impractical. Thus, there is a need for mutation-independent treatment of retinitis pigmentosa.

The Technology: Mutation-independent gene editing therapy for autosomal dominant disease-related genes

This technology is a modified CRISPR/Cas9 gene editing system to overcome limitations of mutation-specific treatment of autosomal dominant disease-related genes. By using a pair of viral vectors, both wild-type and mutated autosomal dominant disease-related genes are removed and replaced by a modified autosomal dominant disease-related gene to restore normal gene function. This technology provides greater long-term efficacy. As such, this simple mutation-independent gene therapy system can treat autosomal dominant diseases including the autosomal dominant disease-related gene in retinitis pigmentosa.

This technology has been validated in a humanized mouse model of autosomal dominant retinitis pigmentosa.

Applications:

• Treatment for autosomal dominant diseases including retinitis pigmentosa
• Treatment for autosomal dominant disorders
• Research tool for gene editing therapies

Advantages:

• Greater long-term efficacy
• Mutation-independent treatment
• Large applicability to autosomal dominant disorders

Lead Inventor:

Stephen Tsang, M.D., Ph.D.

Patent Information:

Patent Pending (US20210017509)

Related Publications:

• Wu WH, Tsai YT, Huang IW, Cheng CH, Hsu CW, Cui X, Ryu J, Quinn PMJ, Caruso SM, Lin CS, Tsang SH. “CRISPR genome surgery in a novel humanized model for autosomal dominant retinitis pigmentosa.” Mol Ther. 2022 Apr 6; 30(4):1407-1420.

Tech Ventures Reference:

• IR CU18283, CU15206

• Licensing Contact: Kristin Neuman