To develop AVR-RD-03, Avrobio leveraged its lentiviral vectors with glycosylation-independent lysosomal targeting-tag technology, which consists of a short peptide sequence linked to a therapeutic protein. The platform is designed to enhance uptake in key tissues and provide a potent transgene promoter to boost protein production.

In the study, Avrobio researchers studied 10 different lentiviral vectors with therapeutic transgenes in mice. Each vector was successful in increasing expression of the GAA, the gene responsible for Pompe disease. The leading vector had minimal off-target effects.

Based on the data, Avrobio will move forward with an investigational new drug (IND)- enabling proof-of-concept study to be completed by the end of the year.

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