Highlights from Retina International World Congress 2024: CRISPR Gene Editing for Inherited Retinal Diseases

CRISPR Medicine News attended the Retina International World Congress 2024 hosted by Irish patient-led charity Fighting Blindness. The theme ‘Targeting the Cure’ emphasized the urgent need for new treatments for inherited retinal dystrophies, which affect one in 4,000 people globally.

The congress featured presentations on various topics such as gene editing by experts like Mark Pennesi MD/PhD and Leah Byrne PhD. Mark Pennesi presented data from Editas Medicine’s Phase 1/2 BRILLIANCE trial of EDIT-101 for Leber congenital amaurosis 10 (LCA10), a disease caused by mutations in the CEP290 gene. The CRISPR-based therapy showed promising results in improving vision in patients.

Leah Byrne discussed her work on developing in vivo models of PRPF31 retinitis pigmentosa, a form of RP that causes vision loss. Her team’s research focused on gene augmentation to treat RP11, with successful results in mouse models. They are now translating these findings to primate models for further study.

The congress highlighted the importance of CRISPR technology in developing new treatments for retinal diseases. The research presented by experts like Mark Pennesi and Leah Byrne offers hope for patients with inherited retinal dystrophies. Stay tuned for more updates on CRISPR Medicine News.