Sangamo Therapeutics publishes ST-506 nonhuman primate brain presentation

Sangamo Therapeutics, Inc.

Sangamo Therapeutics, Inc.

SGMO

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  • Sangamo Therapeutics published a research presentation on ST-506, a blood-brain barrier-penetrant AAV gene therapy designed to repress prion protein expression by targeting PRNP in neurons.
  • Nonhuman primate brain data at 28 days post-dose showed 33.9% of superior frontal gyrus neurons classified as ZFR+ versus 0.29% for vehicle.
  • Single-cell analysis showed PRNP expression reduced by more than 95% in ST-506-expressing neurons, with median PRNP spot total intensity down 21.1% in ZFR+ neurons versus vehicle.
  • Program update cited tolerability in nonhuman primates at 1E+14 vg/kg over 28 days, supporting further clinical development in symptomatic prion disease.


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