AVROBIO announces clinical and regulatory progress for Gaucher Disease Program
Singapore, Jan. 17 -- US based AVROBIO, a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, 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.
"Many people with Gaucher disease type 1 experience life-limiting symptoms even while on chronic enzyme replacement th...
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