A groundbreaking clinical trial called BRILLIANCE has shown promising results in using CRISPR gene editing to treat inherited blindness. Led by Dr. Eric Pierce from Mass Eye and Ear, the study involved 14 participants with Leber Congenital Amaurosis caused by mutations in the CEP290 gene.
The participants, ranging from ages 10 to 63, received a single injection of a CRISPR-based medicine called EDIT-101 directly into one eye. This marked the first time CRISPR was administered inside the body. The trial focused on safety and found no serious adverse events related to the treatment.
More importantly, 11 out of the 14 participants experienced improvements in their vision. This was a significant breakthrough, considering these individuals had no other treatment options prior to the trial. Some reported being able to see things they hadn’t seen before, like food on their plates.
The improvements were measured through various tests, including visual acuity, full-field stimulus testing, visual function navigation, and vision-related quality of life. Several participants showed meaningful enhancements in these measures, indicating the potential of CRISPR gene therapy to correct genetic defects and improve vision in those with inherited retinal disorders.
Overall, the findings from the BRILLIANCE trial highlight the safety and efficacy of CRISPR gene editing in treating inherited blindness, offering hope for those with previously untreatable conditions. Further research is needed to better understand who might benefit most from this innovative therapy.
