Editas Medicine announced clinical data from the Phase I/II BRILLIANCE trial of EDIT-101—an in vivo CRISPR/Cas9 genome editing medicine to treat Leber congenital amaurosis 10 (LCA10). Although the genome editing medicine demonstrated a favorable safety profile across all dose cohorts, the small population of responders will lead the company to pause enrollment in the trial. Editas will seek to identify a collaboration partner to continue development of EDIT-101.
“The results from the BRILLIANCE trial provide a proof of concept and important learnings for our inherited retinal disease programs. We’ve demonstrated that we can safely deliver a CRISPR-based gene editing therapeutic to the retina and have clinically meaningful outcomes,” said Gilmore O’Neill, president and CEO, Editas Medicine. “While we will not progress EDIT-101 on our own and have made the decision to pause enrollment, we have the patient community top of mind and are looking for a collaboration partner to advance this program.”
LCA is the most common cause of inherited childhood blindness, with an incidence of approximately three per 100,000 live births worldwide. Symptoms of LCA appear within the first years of life, resulting in significant vision loss and potential blindness. The most common form of the disease, LCA10, is a monogenic disorder caused by mutations in the CEP290 gene and is the cause of disease in approximately 20–30% of all LCA patients. There is no effective treatment currently available for this disease.
EDIT-101 is designed to treat LCA by deleting the IVS26 CEP290 mutant allele. EDIT-101 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells.
The BRILLIANCE update includes safety and efficacy data from all 14 patients treated in the study to date, which includes 12 adult patients and two pediatric patients. Three out of 14 treated subjects met a responder threshold having experienced clinically meaningful improvements in best corrected visual acuity (BCVA) and demonstrated consistent improvements in two of the following three additional endpoints.
An examination of baseline characteristics of the treatment responder patients revealed that two of the three responders were homozygous for IVS26 mutation (100% of the homozygous patients treated). No other baseline characteristics that could pre-select a responder patient population were identified in the BRILLIANCE dataset.
Since LCA10 patients homozygous for CEP290 IVS26 mutation represent an estimated population of approximately 300 in the United States, Editas will not progress this program independently, and will seek to identify a collaboration partner to continue the development of EDIT-101. Therefore, Editas Medicine will pause further enrollment in the BRILLIANCE trial and will continue long-term follow-up of all patients who have been treated to date.