CAMBRIDGE, Mass., Dec. 19, 2018 (GLOBE NEWSWIRE) -- AVROBIO, Inc. (NASDAQ:AVRO) (the "Company"), a Phase 2 clinical-stage gene therapy company developing gene therapies to potentially cure rare diseases with a single dose, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for the Company's investigational gene therapy, AVR-RD-01, for the treatment of Fabry disease. The gene therapy consists of the patient's own hematopoietic stem cells transduced with AVROBIO's lentiviral vector, carrying a functional version of the GLA gene that encodes α‑galactosidase A (AGA) – the enzyme that is deficient in Fabry disease. Patients with Fabry disease are currently enrolling in AVRO-RD-01-201, the Company's Phase 2 clinical trial (FAB-201), as well as in an investigator-sponsored Phase 1 study.
"Receiving orphan-drug designation from FDA is a positive step forward in our global strategy to advance single-dose, gene therapy as a new treatment paradigm for Fabry disease. We see a promising opportunity for gene therapy to displace today's enzyme replacement therapies that currently leave significant disease progression, a shortened lifespan and other unmet medical needs for patients with Fabry disease," said Geoff MacKay, President and CEO of AVROBIO. "Working with medical leaders, experts and patient advocates, we are committed to moving forward with the clinical and commercial development of our gene therapies because of the potentially dramatic benefit a single-dose therapy can have in transforming the lives of patients."
Orphan-drug designation is granted by the FDA to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan-drug designation provides certain incentives which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers.
About AVR-RD-01
AVR-RD-01 is an ex vivo lentiviral gene therapy being investigated as a single-dose therapy with the potential to provide durable and life-long potential therapeutic benefit for patients with Fabry disease. AVR-RD-01 is designed to employ a state-of-the-art lentiviral vector platform that is an efficient and proven gene transfer system for the integration of a functional copy of the gene into the patient's own stem cells. In patients with Fabry disease, hematopoietic stem cells are collected from the patient, and then transduced with lentiviral vector carrying a functional version of the GLA gene that encodes α‑galactosidase A (AGA) – the enzyme that is deficient in Fabry disease – to create AVR-RD-01 gene therapy. AVR-RD-01 is then infused back into the patient with the goal of restoring normal GLA gene expression such that functional AGA enzyme is sufficiently produced by the patient's own body.
About AVROBIO, Inc.
Forward-Looking Statements
Investor Contact: Christopher F. Brinzey Westwicke Partners 339-970-2843 [email protected] Media Contact: Kathryn Morris The Yates Network Tel: 914-204-6412 [email protected]
© 2026 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
To add Benzinga News as your preferred source on Google, click here.
