Avrobio launches gene therapy program for Gaucher disease

Cambridge, Mass.-based Avrobio said today that it expanded its pipeline to include a preclinical program focused on a gene therapy for Gaucher disease.

The investigational gene therapy makes use of a patient’s blood stem cells, genetically modifying them with a fully functional copy of the faulty gene responsible for the glucosylceramide accumulation that is characteristic of Gaucher disease. Avrobio licensed the technology from Lund University’s Dr. Stefan Karlsson, but did not disclose the terms of the agreement.

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