Avrobio, Inc. Announces Clinical and Regulatory Progress for Gaucher Disease Program
January 14, 2020 at 12:00 pm
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AVROBIO, Inc. announced that it has received notice of clearance from the U.S. Food and Drug Administration (FDA) regarding an Investigational New Drug (IND) application for AVR-RD-02, its investigational gene therapy for the treatment of Gaucher disease. This follows receipt of FDA orphan drug designation status for AVR-RD-02, and now clears AVROBIO to expand its ongoing Phase 1/2 clinical trial in Gaucher disease to the United States, supported by the Company’s proprietary plato gene therapy platform. The standard of care for Gaucher disease type 1 is enzyme replacement therapy (ERT), which often does not halt disease progression or adequately address common life-limiting symptoms reported by Gaucher disease type 1 patients on ERT, such as fatigue and bone pain. Unlike ERT, AVR-RD-02 aims to provide a therapeutic benefit both systemically and throughout the central nervous system (CNS). AVROBIO believes AVR-RD-02 could slow, halt or potentially reverse symptoms throughout the entire body and brain, such as GBA-related Parkinson’s disease which occurs more frequently in people with Gaucher disease type 1. AVROBIO has received consent from the first patient to enroll in GAU-201, a global Phase 1/2 trial of AVR-RD-02. The trial is actively recruiting in Australia and Canada, with additional sites planned in the United States. It is intended to recruit 8 to 16 patients between the ages of 16 and 35 with Gaucher disease type 1, including both those who are treatment-naïve and those who are stable on enzyme replacement therapy. AVROBIO’s gene therapy platform, plato, has now been cleared by regulators in Canada, Australia and the United States for use in the AVR-RD-02 clinical program.
AVROBIO, Inc. is a gene therapy company. The Company is focused on developing potentially curative hematopoietic stem cells (HSC) gene therapies to treat patients with rare diseases following a single dose treatment regimen. The gene therapies that the Company is engaged in developing employ HSCs that are harvested from the patient and then modified with a lentiviral vector to insert the equivalent of a functional copy of the gene that is mutated in the target disease. The Company's development focus has been on a group of rare genetic diseases referred to as lysosomal disorders, primarily managed with enzyme replacement therapies (ERTs). Its pipeline is comprised of three HSC gene therapy programs, which include AVR-RD-02 for the treatment of Gaucher disease type 1 and type 3; AVR-RD-03 for the treatment of Pompe disease, and AVR-RD-01 for the treatment of Fabry disease.