Avrobio is set to incorporate its plato platform into its FAB-201 Phase II trial and dose patients at clinical sites in the US.
The move comes after the US Food and Drug Administration (FDA) cleared the company’s investigational new drug application for its gene therapy candidate AVR-RD-01 to treat Fabry disease.
The plato platform comprises a four-plasmid vector system, automation of a closed cell manufacturing process and a conditioning regimen that uses therapeutic drug monitoring.
Avrobio president and CEO Geoff MacKay said: “We are very pleased that FAB-201 remains on track to expand into sites in the US in the second half of 2019.
“Importantly, we believe this US FDA clearance represents a major milestone as we transition to plato, our optimised commercial-scale platform for our anticipated future worldwide commercialisation activities.
“We have now achieved initial regulatory clearances for clinical trials in Australia, Canada, and the US which incorporate our plato platform.”
The company has dosed seven patients with AVR-RD-01 across the investigator-sponsored Phase I clinical study and FAB-201 and the Phase II clinical trial in Fabry disease currently underway in Australia.
Additional preliminary clinical data from these trials is expected to be available this year.
The open-label, multinational FAB-201 study will evaluate the efficacy and safety of AVR-RD-01 in about eight to 12 treatment-naïve males, aged 16 years and older with Fabry disease.
During the second half of this year, the study will expand into the US and includes screening, baseline, pre-transplant, transplant, and post-transplant follow-up at 48 weeks.
After completion of the study, consenting subjects will continue periodic safety and efficacy assessments for about 14 years.