Results from a groundbreaking clinical trial of CRISPR gene editing in 14 individuals with a form of inherited blindness show that the treatment is safe and led to measurable improvements in 11 of the participants treated.
May 6 2024Mass Eye and Ear The phase 1/2 trial called BRILLIANCE, was led by principal investigator Eric Pierce, MD, PhD, of Mass Eye and Ear , a member of the Mass Gene ral Brigham healthcare system, and sponsored by Editas Medicine , Inc. Findings are reported May 6th in The New England Journal of Medicine .
All 14 trial participants, including 12 adults and two children , were born with a form of Leber Congenital Amaurosis caused by mutations in the centrosomal protein 290 gene. They underwent a single injection of a CRISPR/Cas9 genome editing medicine, EDIT-101 in one eye via a specialized surgical procedure.
"The results from the BRILLIANCE trial provide proof of concept and important learnings for the development of new and innovative medicines for inherited retinal diseases. We've demonstrated that we can safely deliver a CRISPR-based gene editing therapeutic to the retina and have clinically meaningful outcomes," said Baisong Mei, MD, PhD, Chief Medical Officer, Editas Medicine.
CRISPR-Cas9 is a gene editing toolkit that acts as a GPS-guided scissor to cut a portion of the mutated genome to leave a functional gene. For inherited blindness, the goal was to inject CRISPR to reach the eye's retina to restore the ability to produce the gene and protein responsible for light-sensing cells.
"This trial shows CRISPR gene editing has exciting potential to treat inherited retinal degeneration," Pennesi said. "There is nothing more rewarding to a physician than hearing a patient describe how their vision has improved after a treatment. One of our trial participants has shared several examples, including being able to find their phone after misplacing it and knowing that their coffee machine is working by seeing its small lights.
Clinical Trial Gene Cas9 Children CRISPR Ear Efficacy Eye Food Gene Therapy Gene-Editing Genetic Genome Genome Editing Genomics Healthcare Hearing Hospital Laboratory Leber Congenital Amaurosis Medical School Medicine Neck Ophthalmology Protein Research Retinal Degeneration Throat Vision Loss
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