Here's Why Editas Stock Dropped After Data From CRISPR-Based Retinal Disorder Candidate

  • Editas Medicine Inc EDIT has announced initial clinical data from the ongoing Phase 1/2 BRILLIANCE trial of EDIT-101 for blindness due to Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder. 
  • Related Link: Editas Medicine Unveils Preclinical Data From New Gene Editing Technology.
  • Preliminary results include safety and efficacy data from the first two cohorts, the adult low-dose cohort (6x1011 vg/ml) and the adult mid-dose cohort (1.1x1012 vg/ml). 
  • Safety data were reported for all six subjects treated in the low dose (n=2) and mid-dose (n=4) cohorts. 
  • Most adverse events (AEs) were mild and primarily resulted from the surgical procedure and subretinal injection. There were no dose-limiting toxicities (DLTs). 
  • Investors are probably reacting to mild anterior chamber inflammation observed in patients and controlled with oral steroids. 
  • No Cas9-specific antibody or T-cell response was detected. 
  • To date, no treatment-related cataracts, edema, or retinal thinning have been observed.
  • Two of three subjects in the mid-dose cohort followed for up to six months showed efficacy signals suggesting productive editing and providing initial support for clinical benefits, including improvements in BCVA, FST, and/or mobility navigation.
  • In June, the Independent Data Monitoring Committee (IDMC) endorsed proceeding with the first pediatric cohort to review clinical safety data from the adult low-dose and mid-dose cohorts. 
  • Treatment in the adult high-dose cohort continues, and the pediatric mid-dose cohort is commencing.
  • Price Action: EDIT stock is down 20.50% at $41.71 during the market session on the last check Wednesday.
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