Gene Editing, Results

Sangamo shares take a big hit as investors react to setback for pioneering gene-editing tech

Sangamo {$SGMO} today released the first snapshots from two early-stage studies looking at the effect their first-generation gene editing technology has on two rare and lethal diseases — MPS I and II. And from it Sangamo CEO Sandy Macrae has reached two basic conclusions:

“It’s working and it’s not sufficient for a clinical benefit.”

That conclusion — along with Sangamo’s assertion that their zinc finger nuclease approach is safe — is driving a switch-up in the clinic, where the biotech is pursuing a historic attempt to engineer a breakthrough in gene-editing humans after years of animal research.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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