- 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.
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
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