A recent clinical trial has shown promising results for a CRISPR/Cas9 gene editing therapy that could potentially restore vision in individuals with a rare type of inherited or congenital blindness.
The trial, called BRILLIANCE, involved 14 participants - 12 adults and 2 children - who received treatment with EDIT-101 to address Leber Congenital Amaurosis (LCA) caused by mutations in the CEP290 gene. The trial is seen as a landmark achievement in the treatment of genetic blindness, offering an alternative treatment option when traditional forms of gene therapy are not viable.
During the trial, each participant received an injection of EDIT-101 in one eye through a surgical procedure. The participants were divided into three groups receiving different doses of the therapy, with children receiving a mid-dose treatment. Over 12 months, participants were monitored every three months for safety, with follow-up continuing for an additional two years.
The results revealed no severe adverse events or toxicities related to the treatment, indicating that CRISPR-based therapy can be safely delivered to the retina with clinically significant results.
While full vision restoration was not achieved, some individuals experienced improvements in their visual abilities, with a few managing to read certain lines on an eye chart. The outcomes of the BRILLIANCE trial were seen as pivotal for advancing treatments for inherited retinal diseases.
Editas Medicine, the company that initially funded the BRILLIANCE trial, paused enrollment in November 2022, leading the researchers to explore collaborations with other commercial partners for further trials.
Dr. Eric Pierce, the lead author of the study, expressed optimism that the published results would attract interest from the biotech and pharmaceutical sectors to support Phase 3 studies.
Future investigations aim to delve into optimal dosing strategies, potential age-related treatment effects, and improved outcome measures for evaluating the therapy's impact on daily activities.
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