CAMBRIDGE, Mass.–(BUSINESS WIRE)–AVROBIO (NASDAQ: AVRO) (the “Company”), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the first patient has been dosed using the Company’s plato™ gene therapy platform, which is intended to provide the foundation for the potential worldwide commercialization of AVROBIO’s gene therapies.

“Our plato platform sets AVROBIO apart. Early in AVROBIO’s growth, we prioritized the development of plato to address and surmount key challenges facing gene therapy, and believe we have made great strides towards that goal”

Tweet this

The plato platform includes a state-of-the-art lentiviral vector (LV2) designed to optimize vector copy number, transduction efficiency and resulting enzyme activity; a personalized conditioning regimen with precision dosing via therapeutic drug monitoring intended to enhance safety and engraftment; advanced cryopreservation to extend shelf life and enable flexible scheduling for patients; and an automated, closed-manufacturing process intended to improve consistency and predictability of the drug product. The Company expects that plato’s self-contained manufacturing pods will facilitate global manufacturing site expansion.

The first patient dosed using plato is enrolled in FAB-201, the Company’s Phase 2 trial of AVR-RD-01, an investigational gene therapy for Fabry disease. That trial is evaluating the safety and efficacy of AVR-RD-01 in 8 to 12 treatment-naïve patients, with ongoing recruitment in the U.S., Canada and Australia.

“Our plato platform sets AVROBIO apart. Early in AVROBIO’s growth, we prioritized the development of plato to address and surmount key challenges facing gene therapy, and believe we have made great strides towards that goal,” said Geoff MacKay, AVROBIO’s President and Chief Executive Officer. “We have delivered improvements in manufacturing across the supply chain and into the clinic. We are proud to say that our gene therapy programs are powered by plato.”

plato has received regulatory clearance for clinical use and will be used for all patients going forward in FAB-201, AVROBIO’s Phase 2 Fabry disease trial, and in GAU-201, AVROBIO’s Phase 1/2 clinical trial of AVR-RD-02, an investigational gene therapy for Gaucher disease.

About AVROBIO

AVROBIO’s mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease progression throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we are also advancing a program in Pompe disease. AVROBIO is powered by plato™, our streamlined platform for worldwide gene therapy commercialization. We are headquartered in Cambridge, Mass. with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.