AVROBIO receives orphan-drug designation from FDA for AVR RD 02 to treat Gaucher disease

This article was originally published here

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. The Company is actively recruiting

The post AVROBIO receives orphan-drug designation from FDA for AVR RD 02 to treat Gaucher disease appeared first on Pharmaceutical Business review.

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