With a potential cure for lysosomal storage disorders in hand, Avrobio raises $60M to back gene therapy pipeline
In gene therapy, responses from just one or two patients can have a huge influence on analysts’ opinions. Just ask the executive team at bluebird bio, which trumpets each patient’s progress with delight — or calibrates a new development strategy when smiles turn to frowns.
So it’s no wonder that Avrobio is so proud of its very first patient.
At a scientific conference in Japan last fall, researchers reviewed the case of a patient with Fabry disease whose blood levels of the enzyme a-Gal A were near zero at the start of treatment. Within 45 days of receiving their gene therapy AVR-RD-01, the patient’s plasma a-Gal A activity increased into the normal range, they said. And 6 months later that hadn’t changed.
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