Gene editing to treat retinal dystrophies

This technology is a gene editing methodology for correcting mutations in the CRB1 gene, one of the major cause of inherited retinal dystrophies.

Unmet Need: Treatment modality available to retinal dystrophy patients with mutations in the CRB1 gene

Current methods to treat CRB1-linked inherited retinal dystrophies focus on a gene augmentation approach. Attempts to augment CRB1 protein level have been unsuccessful due to the diversity of retinal CRB1 isoforms and their expression in different cell types of the retina. Although more precise gene editing tools are being developed, there is no therapy commercially available to target specifically CRB1 mutations.

The Technology: Efficient gene editing tool for correcting CRB1 mutations in patients with inherited retinal dystrophies

This technology aims to use prime gene editing to correct CRB1 mutations in patients with retinal dystrophies. Prime editing has emerged as an improved gene editing alternative being able to correct transition and transversion mutations in addition to small insertions and deletions. It is used here to specifically correct prevalent mutations in CRB1 gene in patient-derived iPSCs.

This technology has been validated in vitro with patient iPSCs.

Applications:

• Treatment for inherited retinal dystrophy due to CRB1 mutations
• Research tool to validate therapeutic efficacy
• Research tool to study retinal dystrophies
• Research strategy to optimize prime editing
• Suitable to be delivered with viral or nanoparticle-based delivery methods.

Advantages:

• Isoform independent method
• Efficiently corrects prevalent mutations in the CRB1 gene
• Can be used for cell therapy
• Can be used as a gene therapy

Lead Inventor:

Peter M.J. Quinn, Ph.D.

Patent Information:

Patent Pending

Tech Ventures Reference:

• IR CU22019

• Licensing Contact: Kristin Neuman