Gene therapy company pivots away from Fabry disease

5 January 2022

A change of direction has spooked investors in Avrobio (Nasdaq: AVRO), as the firm announced “variable” Phase II results in Fabry disease.

Avrobio, which is working on its own lentiviral gene therapy technology, has long been focused on lysosomal storage disorders, including Fabry, Gaucher, and Pompe diseases.

Shares in the firm dropped over a third on Tuesday, as the company announced it was “deprioritizing its Fabry disease program,” due to several factors, including new data showing mixed engraftment patterns for patients in the trial.

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