The FDA has signed off on AVROBIO’s (NASDAQ:AVRO) IND for a Phase 1/2 clinical trial evaluating gene therapy AVR-RD-02 for the treatment of Gaucher disease, an inherited disorder caused by the accumulation of a type of lipid in tissues due to the dysfunction in a certain enzyme.
The primary efficacy endpoint of the 16-subject study is change from baseline in GCase enzyme activity at week 52 post-treatment. The estimated primary completion date is May 2021.
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