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

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.

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