Avrobio is looking to bounce back from an earlier failure with its hematopoietic stem cell (HSC) gene therapy platform, and has just unveiled promising initial data from the first five patients treated for Gaucher’s disease with its investigational candidate, AVR-RD-02.
Gaucher’s disease is the most common lysosomal disorder, affecting 50,000 to 100,000 people worldwide. Characterized by a build up of...
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