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AVROBIO Presents Clinician Experience with FAB-GT Clinical Trial and Updated Safety Data for Investigational Gene Therapies in Fabry Disease and Gaucher Disease Type 1
Safety data updates for Phase 1 and 2 Fabry disease clinical trials and Phase 1/2 Gaucher disease type 1 clinical trial show no adverse events or serious
About this update from Tectonic Therapeutic, Inc.
[{"type":"text","content":"\nSafety data updates for Phase 1 and 2 Fabry disease clinical trials and Phase 1/2 Gaucher disease type 1 clinical trial show no adverse events or serious adverse events related to drug product\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a vision to free people from a lifetime of genetic disease, today hosted a panel of investigators managing the patient experience in the Phase 2 FAB-GT clinical trial of AVR-RD-01, an investigational one-time gene therapy for Fabry disease, at the 14th International Congress of Inborn Errors of Metabolism (ICIEM), Nov. 21-23, 2021, in Sydney, Australia. Other presentations included updated safety data from the first lentiviral gene therapy clinical trials for Fabry disease and Gaucher disease type 1, as well as 10- to 12-month safety data from gene therapy-treated mice with mucopolysaccharidosis type II (MPSII), or Hunter syndrome.\n\nChaired by Rob Wynn, M.D., (consultant pediatric hematologist at the Royal Manchester Children’s Hospital, UK), the AVROBIO sponsored symposium, Advancing the patient experience during ex vivo lentiviral gene therapy for lysosomal disorders, featured trial investigators Mark Thomas, M.D., (Royal Perth Hospital, WA, AUS; clinical professor at the University of Western Australia Medical School), Kathy Nicholls, M.D., (Royal Melbourne Hospital, VIC, AUS) and Ben Carnley, M.D., (Royal Perth Hospital, WA, AUS), who discussed managing the patient experience with ex vivo lentiviral gene therapy based on their involvement in the FAB-GT clinical trial.\n\nDuring the panel discussion, the clinicians discussed the patient experience during apheresis (the process by which the patient’s stem cells are collected), conditioning (the process to clear space in the patient’s bone marrow to receive their stem cells back following introduction of the therapeutic transgene), and infusion of the genetically modified stem cells, as well as after receiving the gene therapy.\n\n“It’s been exciting and rewarding to be part of this first-in-the-world clinical trial to bring this investigational gene therapy to the Fabry disease community here in Australia,” said Dr. Nicholls. “While the current standard of care has made an important difference in the lives of patients, there are limitations in the impact of enzyme...