The company is currently evaluating the gene therapy in a phase I/II clinical trial. AVR-RD-02 has already received orphan drug designation from the U.S. Food and Drug Administration (FDA).

Gaucher disease is a rare, inherited lysosomal storage disorder that results in toxic accumulation of glucosylceramide and glucosylsphingosine in macrophages. It is estimated that 1 in 44,000 people are diagnosed with Gaucher disease.

AVR-RD-02 contains a patient's hematopoietic stem cells that have been genetically modified to express cerebrosidase, the enzyme that is deficient in Gaucher disease. The therapy is delivered using lentiviral vectors, which have a large cargo capacity and the ability to integrate therapeutic genes directly into a patient's chromosomes.

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Avrobio moves forward with gene therapy candidate
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