Abstract
This is the first in human treatment with ST-920, a recombinant AAV2/6 vector encoding the
cDNA for human a-Gal A. The purpose of this study is to evaluate the safety and tolerability
of ascending doses of ST-920. ST-920 aims to provide stable, long-term production of α-Gal A
at therapeutic levels in subjects with Fabry disease. The constant production of α-Gal A in
humans should, importantly, enable reduction and potentially clearance of Fabry disease
substrates Gb3 and lyso-Gb3. On Day 1, patients will be infused intravenously with a single
dose of ST-920 and followed for a period of 52 weeks.
