Editas' CRISPR-based eye therapy proves safe, but the jury's still out on whether it works
Researchers at Editas Medicine revealed long-anticipated data for their CRISPR gene editing program Wednesday morning, the second-ever batch of human results for in vivo administration of the Nobel Prize-winning technology. And while the therapy appeared remarkably safe, a huge step forward in its own right, experts questioned whether the early efficacy figures will ultimately bear fruit.
Editas’ data come from a Phase I/II study evaluating a gene editing candidate for LCA10, a form of inherited blindness, with the first cut looking at six adult patients across two low- and mid-dose cohorts. There were no dose-limiting toxicities and most adverse events stemmed from the surgical procedure used to inject the treatment into the eye, Editas said. It was safe enough for the independent data monitoring committee to rubber stamp testing the experimental drug in children.
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