Benzinga | Mar 1, 2021 07:04AM EST

AVROBIO Receives Orphan Drug Designation From European Commission For AVRRD04, An Investigational Gene Therapy For Cystinosis

AVROBIO, Inc. (NASDAQ:AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced that the European Commission (EC) has granted orphan drug designation for AVR-RD-04, the company's investigational gene therapy for the treatment of cystinosis. AVR-RD-04 consists of the patient's own hematopoietic stem cells, genetically modified to express cystinosin, the protein that is deficient in patients with cystinosis. AVR-RD-04 is currently being evaluated in a Phase 1/2 clinical trial (NCT03897361) sponsored by AVROBIO's academic collaborator at the University of California, San Diego.1