The BRILLIANCE trial, a Phase I/II clinical trial, has shown promising results for the use of CRISPR/Cas9 gene editing therapy in treating a rare form of inherited blindness. Out of the 14 participants in the trial, 79 percent showed measurable improvement after receiving an injection of EDIT-101 into one eye.
The individuals in the trial had a form of Leber Congenital Amaurosis (LCA) caused by mutations in the CEP290 gene. This type of genetic blindness cannot be treated with traditional forms of gene therapy, making this trial a significant breakthrough in the field.
Dr. Tomas Aleman, the principal investigator of the study, commented on the importance of this trial in treating genetic blindness. He stated that this trial represents a landmark in the treatment of genetic blindness, as previous forms of gene therapy were not effective for this condition.
During the trial, participants received different doses of the CRISPR-based gene editing therapy. Two adults received low-dose therapy, five received mid-dose therapy, and five received high-dose therapy. Two children also received a mid-dose of EDIT-101.
The participants were monitored every three months for a period of twelve months to assess the safety of the treatment. The researchers confirmed that follow-up was less frequent for an additional two years. Fortunately, there were no serious adverse effects or toxicities related to the treatment or procedure.
The results of the BRILLIANCE trial have provided proof of concept and important learnings for the development of new treatments for inherited retinal diseases. Dr. Baisong Mei, Chief Medical Officer of Editas Medicine, explained that this trial has demonstrated the safety and effectiveness of CRISPR-based gene editing therapy for retinal diseases.
Looking to the future, further studies are needed to determine ideal dosing and whether certain age groups, such as younger patients, respond better to the treatment. The researchers also mentioned the importance of refining endpoints to measure the effects of improved cone function on daily activities.
In conclusion, the results of the BRILLIANCE trial have shown promising outcomes for the use of CRISPR/Cas9 gene editing therapy in treating a rare form of inherited blindness. This trial has paved the way for future research and development of innovative medicines to treat genetic blindness and other inherited retinal diseases.