AVROBIO’s IND for commercial-scale Fabry disease gene therapy cleared by the FDA

AVROBIO’s (NASDAQ:AVRO) IND for AVR-RD-01 – a gene therapy candidate for the treatment of Fabry disease – has been cleared by the FDA, enabling AVROBIO to use its commercial-scale gene therapy manufacturing system, plato, in clinical trials going forward.

Fabry disease is a genetic lysosomal storage disease in which a mutation in the GLA gene results in an accumulation of lipids in a patients’ cells, impeding normal cellular functions in tissues and organs. AVR-RD-01 is designed to transfer a functional GLA gene into hematopoietic stem cells collected from the patient, which are then reinfused.

“We believe this U.S. FDA clearance represents a major milestone as we transition to plato, our optimized commercial-scale platform for our anticipated future worldwide commercialization activities,” Geoff MacKay, AVROBIO’s president and CEO, said in a statement.

AVROBIO plans to incorporate plato-produced AVR-RD-01 into its ongoing Phase 1/2 trial in Australia, which will expand to the U.S. in the second half of this year.

Sign up for the BioTuesdays weekly newsletter

Get the latest news on the healthcare industry’s corporate and clinical developments, executive moves, and market updates. Every Tuesday, in your inbox.