Citing Small Population, Editas Medicine Pauses Work On Gene Therapy Trial For Genetic Eye Disorder

Editas Medicine Inc EDIT announced clinical data from the Phase 1/2 BRILLIANCE trial of EDIT-101, an in vivo CRISPR/Cas9 genome editing medicine.
EDIT-101 is under development for the treatment of blindness due to Leber congenital amaurosis 10 (LCA10, a CEP290-related retinal degenerative disorder).
It is designed to repair the IVS26 CEP290 mutant allele that impacts approximately 1,500 LCA10 patients in the U.S.
The BRILLIANCE update includes safety and efficacy data from all 14 patients treated in the study to date.
Three out of 14 treated subjects experienced clinically meaningful improvements in best corrected visual acuity (BCVA) and demonstrated consistent improvements in two of the following three additional endpoints: full field sensitivity test (FST), visual function navigation course (VFN), or the visual function quality of life (VFQ).
EDIT-101 was tolerated, with no ocular serious adverse events or dose-limiting toxicities observed.
Given the small population (around 300 in the U.S.), the company will pause enrollment in the BRILLIANCE trial and seek to identify a collaboration partner to continue the development of EDIT-101.
Editas Medicine will continue long-term follow-up of all patients.
Price Action: EDIT shares are down 12% at $10.78 on the last check Thursday.