Sangamo team rushes to defend first human gene editing data as MPS II therapy offers a hit — and a miss
Sangamo $SGMO rolled out its first glimpse at the clinical efficacy of their gene editing therapy for Hunter syndrome (MPS II). And while researchers were able to point to some clear evidence that their approach had an impact on a key biomarker for the disease for 2 patients, there was a critical lack of proof that it was doing specifically what it was designed for.
Understandably, Sangamo concentrated on urinary glycosaminoglycans (GAGs), highlighting big drops for both patients in the second cohort of the tiny exploratory study.
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