Avrobio gene therapy eliminates toxic substrate in Fabry patient

The first Fabry disease patient treated with Avrobio’s plato gene therapy platform in a phase 2 trial has experienced the complete clearance of toxic kidney substrate.

Avrobio designed the plato platform to optimize vector copy number, personalize the conditioning regimen, facilitate automated manufacturing and otherwise improve and industrialize its lentiviral vector-based approach to the treatment of genetic diseases. Late in 2019, a Fabry patient in a phase 2 clinical trial of ex vivo lentiviral gene therapy AVR-RD-01 became the first patient dosed using plato.