An innovative gene-editing technique could potentially improve vision in individuals suffering from an inherited form of blindness, according to a recent publication in the New England Journal of Medicine. This pioneering technique, known as CRISPR gene editing, was tested on a group of clinical trial volunteers with encouraging results.
Roughly 79% of the participants in this clinical trial witnessed a substantial enhancement in their vision after undergoing the CRISPR-based gene editing, which is engineered to correct a rare type of blindness. “This trial demonstrates the immense potential of CRISPR gene editing in treating inherited retinal degeneration,” said Mark Pennesi, M.D., Ph.D., a leading author of the study and an ophthalmologist at Oregon Health & Science University.
The trial, titled BRILLIANCE, was designed to assess the safety and efficacy of EDIT-101, a new gene-editing treatment developed by Editas Medicine. The treatment aims to correct a mutation in the CEP290 gene, which is critical for vision. This mutation results in a rare condition known as Leber Congenital Amaurosis, or LCA, Type 10, a disorder currently lacking FDA-approved treatments.
In early 2020, the first participant was treated at the OHSU Casey Eye Institute, marking the inaugural use of CRISPR for in vivo gene editing. The trial included 14 participants, each receiving EDIT-101 in one eye. The study discovered that 79% of participants showed improvement in at least one of four measured outcomes, while 43% reported improved vision-related quality of life.
Despite the promising results, further research is required. The trial was paused in late 2022, with Editas Medicine seeking another partner to continue the therapy’s development. The researchers are hopeful that future studies will determine the ideal dosage, the age groups that benefit most, and will include refined endpoints to measure impacts on daily activities.
The study represents a significant milestone in the treatment of genetic diseases, specifically genetic blindness, by offering an alternative treatment when traditional therapies, such as gene augmentation, are not an option. The results from the BRILLIANCE trial provide proof of concept and critical insights for the development of innovative medicines for inherited retinal diseases.
The trial was conducted across five clinical sites, including the OHSU Casey Eye Institute, Bascom Palmer Eye Institute, Mass Eye and Ear, Scheie Eye Institute at the University of Pennsylvania and Children’s Hospital of Philadelphia, and Kellogg Eye Center. The groundbreaking research and positive outcomes of these trials underscore the potential of gene-editing technologies in revolutionizing treatments for inherited retinal disorders and blindness.
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