Avrobio Plots Comeback With Early Data From Gaucher’s Gene Therapy
Sees Bluebird Success As Creating Pathway
Early data in Gaucher disease suggest Avrobio’s hematopoietic stem cell gene therapy could help patients who do not response to enzyme replacement therapy.
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The lentiviral gene therapy company is still trading below cash, but the cystinosis results add extra momentum to its comeback.
Bluebird bio’s Tom Klima told Scrip launch preparations are well under way, including negotiations with payers on outcomes-based reimbursement agreements.
Avrobio is sidestepping market and regulatory issues in Fabry disease by suspending development of AVR-RD-01, but says it remains focused on advancing treatment of lysosomal storage disorders with gene therapies.