AVRO - Avrobio' gene therapy for Gaucher disease an Orphan Drug in Europe

Avrobio' (AVRO) AVR-RD-02, a gene therapy for the treatment of Gaucher disease, has been granted Orphan Drug status by the European Commission.AVR-RD-02 consists of patient’s genetically modified hematopoietic stem cells, to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease.The company recently dosed the first patient in the GuardOne Phase 1/2 trial to evaluate the safety and efficacy of AVR-RD-02.Last year, AVR-RD-02 received FDA orphan drug designation for the same indication.

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Avrobio' gene therapy for Gaucher disease an Orphan Drug in Europe