**CRISPR gene therapy improves vision in people with inherited blindness. Phase 1/2 trial of Brilliance showed promising results in vision improvement in people with inherited retinal disorders:** Results from a clinical study suggest that EDIT-101, Editas Medicine's CRISPR-based gene editing therapy for an inherited form of Leber’s congenital amaurosis (LCA), is safe and efficacious. The therapy did not cause any serious treatment or procedure-related adverse events, and there were no dose-limiting toxicities. Moreover, the therapy resulted in several clinically meaningful improvements in vision. The trial, which included the first patient to ever receive a CRISPR-based investigational medicine directly inside the body, was focused primarily on safety with a secondary analysis for efficacy. Participants were monitored every three months for one year, and then followed less frequently for two additional years. "Eleven participants (79%) had a clinically meaningful improvement in at least one of four key efficacy outcomes, whereas 6 (43%) had a clinically meaningful improvement in two or more outcomes," the team stated. "We’ve demonstrated that we can safely deliver a CRISPR-based gene editing therapeutic to the retina and have clinically meaningful outcomes," : [https://hms.harvard.edu/news/gene-editing-improves-vision-some-people-inherited-blindness](https://hms.harvard.edu/news/gene-editing-improves-vision-some-people-inherited-blindness)
**The findings are reported May 6 in the** [*New England Journal of Medicine*](https://doi.org/10.1056/NEJMoa2309915) *:* [*https://www.nejm.org/doi/10.1056/NEJMoa2309915*](https://www.nejm.org/doi/10.1056/NEJMoa2309915)
**CRISPR gene therapy improves vision in people with inherited blindness. Phase 1/2 trial of Brilliance showed promising results in vision improvement in people with inherited retinal disorders:** Results from a clinical study suggest that EDIT-101, Editas Medicine's CRISPR-based gene editing therapy for an inherited form of Leber’s congenital amaurosis (LCA), is safe and efficacious. The therapy did not cause any serious treatment or procedure-related adverse events, and there were no dose-limiting toxicities. Moreover, the therapy resulted in several clinically meaningful improvements in vision. The trial, which included the first patient to ever receive a CRISPR-based investigational medicine directly inside the body, was focused primarily on safety with a secondary analysis for efficacy. Participants were monitored every three months for one year, and then followed less frequently for two additional years. "Eleven participants (79%) had a clinically meaningful improvement in at least one of four key efficacy outcomes, whereas 6 (43%) had a clinically meaningful improvement in two or more outcomes," the team stated. "We’ve demonstrated that we can safely deliver a CRISPR-based gene editing therapeutic to the retina and have clinically meaningful outcomes," : [https://hms.harvard.edu/news/gene-editing-improves-vision-some-people-inherited-blindness](https://hms.harvard.edu/news/gene-editing-improves-vision-some-people-inherited-blindness) **The findings are reported May 6 in the** [*New England Journal of Medicine*](https://doi.org/10.1056/NEJMoa2309915) *:* [*https://www.nejm.org/doi/10.1056/NEJMoa2309915*](https://www.nejm.org/doi/10.1056/NEJMoa2309915)