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Avrobio stops work on rare disease gene therapy after unexpected study results

January 4, 2022

Fabry is one of several dozen diseases known collectively as lysosomal storage disorders, all caused by genetic breakdowns in how the body’s cells process complex fats or sugars. Treatment, when it’s available, usually involves infusions of the digestive proteins that people with these illnesses are missing.

More recently, several lysosomal storage disorders, among them Fabry, have become the target of a cluster of biotech companies that aim to address the genetic cause of these conditions more directly.

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