AVROBIO Receives Rare Pediatric Disease Designation from the U.S. FDA for First-in-Class Gene Therapy for Gaucher Disease

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Oct 27, 2022

AVROBIO%2C+Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to AVR-RD-02, a first-in-class investigational gene therapy that genetically modifies patients’ own hematopoietic stem cells (HSCs) to treat Gaucher disease, a rare lysosomal disorder that can lead to multiorgan pathology, clinical morbidity and early mortality.