Editas has provided first in-human results for its CRISPR candidate for retinal degenerative disorder Leber congenital amaurosis 10 (LCA10), noting some early efficacy signals. Most adverse events seen in the reported low and mid-dose cohorts were mild and primarily resulting from the subretinal injection delivery procedure. Mild anterior chamber inflammation was observed, and controlled with oral steroids.
An analyst from RBC Capital noted that the slim improvement in vision fell short of what ProQR has reported in its sepofarsen (antisense) LCA10 trial. Concern was also raised that the mild anterior chamber inflammation seen could become an issue as dosing is escalated
https://www.fiercebiotech.com/biote...diting-first-human-results-for-edit-101-lca10
https://ir.editasmedicine.com/news-...ounces-positive-initial-clinical-data-ongoing
https://ir.editasmedicine.com/events-and-presentations
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