AVROBIO Receives Rare Pediatric Disease Designation from the U.S. FDA for First-in-Class Gene Therapy for Gaucher Disease
FDAs Rare Pediatric Disease Designation and Voucher Program is intended to facilitate the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
- FDAs Rare Pediatric Disease Designation and Voucher Program is intended to facilitate the development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.
- AVROBIOs Gaucher disease program includes Gaucher disease type 1 and type 3, which collectively impact an estimated 30,000 patients worldwide.
- Gaucher disease is a rare, inherited lysosomal disorder characterized by the toxic accumulation of glucosylceramide (GlcCer) and glucosylsphingosine (GlcSph) in macrophages.
- Our first-in-class pipeline includes clinical programs for cystinosis and Gaucher disease type 1, as well as preclinical programs for Gaucher disease type 3, Hunter syndrome and Pompe disease.