Description

A Phase I, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-913, a rAAV2/6-based Gene Transfer in Subjects with Mucopolysaccharidosis II (MPS II)
StatusFinished
Effective start/end date6/16/175/1/19

Funding

  • Sangamo Therapeutics, Inc.

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Mucopolysaccharidosis II
Safety
Genes