An experimental CRISPR-based treatment from Editas Medicine led to meaningful improvements in the functional vision of a single patient born with a rare, genetic disease that leads to blindness — a preliminary study outcome that Editas called encouraging but that also raises some concerns its gene-editing approach is not potent enough.
The first clinical data from Editas’ treatment, called EDIT-101, were presented Wednesday at a research meeting. One patient out of four treated with a middle dose of EDIT-101 showed meaningful improvements across several different measures of functional vision. A low dose of the treatment tested in two patients was ineffective.
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