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AVROBIO Receives Orphan Drug Designation from the European Medicines Agency for AVR-RD-02, an Investigational Gene Therapy for Gaucher Disease

Monday, September 28, 2020

AVROBIO, Inc., a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, announced that the European Commission (EC) has granted orphan drug designation for AVR-RD-02, the company’s investigational gene therapy for the treatment of Gaucher disease. AVR-RD-02 consists of the patient’s own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease. AVROBIO recently dosed the first patient in the GuardOne Phase 1/2 clinical trial to evaluate the safety and efficacy of AVR-RD-02.

“Like many lysosomal disorders, Gaucher disease can lead to debilitating complications throughout the body and brain. The standard of care does not address all these symptoms and may not be able to halt progression of the disease,” said Geoff MacKay, AVROBIO’s president and CEO. “Our investigational gene therapy is designed to address the head-to-toe manifestations of Gaucher disease with a single dose. We’re pleased to receive orphan drug designation, which recognizes the potential of our approach to transform the standard of care – and, we hope, the quality of life – for people living with this rare genetic disorder.”

The EC grants orphan drug designation to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Orphan drug designation gives companies certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and 10 years of market exclusivity following regulatory approval.

AVR-RD-02 has also received orphan drug designation from the U.S. Food and Drug Administration.

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