Tag Archives: lysosomal storage disorders

September, 2018

September, 2017

  • 22 September

    Avrobio Expands Rare Disease Pipeline with Gene Therapy to Treat Pompe Disease

    CAMBRIDGE, Mass.–(BUSINESS WIRE)–AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to Pompe disease. This pre-clinical program becomes AVROBIO’s third gene therapy for Lysosomal Storage Disorders (LSDs), following on the heels of the Company’s Phase 1 Fabry program …

February, 2017

February, 2016

April, 2015

  • 8 April

    FDA Accepts ArmaGen’s IND for its Hurler Syndrome Candidate

    Privately-held biotech ArmaGen, Inc. announced today that US health regulators have accepted for review its investigational New Drug (IND) application for its drug for a rare lysosomal storage disease. The company, focused on developing novel therapies to treat severe neurological disorders, said that the US Food and Drug Administration (FDA) …