BioCentury
ARTICLE | Clinical News

Vector copy number declines over time in four Fabry disease patients who received Avrobio gene therapy

October 5, 2018 7:42 PM UTC

Avrobio Inc. (NASDAQ:AVRO) said peripheral blood average vector copy number (VCN) declined over time in four Fabry disease patients who received a single dose of gene therapy AVR-RD-01 in either a Phase I or Phase II trial.

AVR-RD-01 comprises CD34+ hematopoietic stem cells transduced ex vivo with a lentivirus to express α galactosidase A (AGA) (see “Aiming for Durability”)...

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Alpha galactosidase A