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Editas Medicine, one of the small handful of original CRISPR companies, announced Thursday it is halting development on its first clinical program after data showed only a small subset of patients were responding.

Edit-101, an experimental CRISPR-based treatment for a rare eye disease called CEP290-meditated LCA10, led to “clinically meaningful” improvements in sight in only three out of 14 patients treated in the company’s Phase I study.


Editas said the only indication for why the three patients responded when most didn’t was that two of them had two — as opposed to one — copies of the mutation that drives the disease. But the company said only around 300 patients matched the genetic profile, too small a market to be commercially viable.

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