Clinical trial design, supply chain and operations

July 2021

  • Challenges in the gene therapy of bone marrow failure syndromes

    J Bueren
    Juan Bueren
    Director of the Biomedical Innovative Unit at the CIEMAT, IIS Fundación Jiménez Díaz and Biomedical Network Centre on Rare Diseases, Madrid, Spain and Vice-President, European Society for Cell and Gene Therapy
    Juan Bueren is the Head of the Division of Hematopoietic Innovative Therapies of CIEMAT and CIBER of Rare Diseases. Dr Bueren is also Coordinator of the Mixed Unit of Advanced Therapies CIEMAT/IIS-Jiménez Díaz Foundation and consultant of Rocket Pharmaceuticals Inc, and currently serves as Vice-President of the European Society for Cell and Gene Therapy. Dr Bueren is Scientific Director of gene therapy trials for patients with Fanconi anemia and also for patients with the primary immunodeficiency, leukocyte adhesion deficiency type I, and has participated in the development of therapeutic lentiviral vectors designed as Orphan Drugs by the European Medicines Agency and by the FDA for these two diseases. Both ODs have been licensed to Rocket Pharmaceuticals which is currently developing different global gene therapy programs in Europe and in the USA for Fanconi anemia and LAD-I.
    16 July 2021
  • Learning lessons from the long, troubled history of stem cell therapy for future clinical success

    J Rasko
    John EJ Rasko
    Director, Department of Cell and Molecular Therapies at Royal Prince Alfred Hospital and the Gene and Stem Cell Therapy Program at the Centenary Institute, University of Sydney
    John EJ Rasko is an Australian pioneer in the application of adult stem cells and genetic therapy. Since 1999 he has directed the Department of Cell and Molecular Therapies at Royal Prince Alfred Hospital and the Gene and Stem Cell Therapy Program at the Centenary Institute, University of Sydney. John Rasko is a clinical hematologist, pathologist and scientist with an international reputation in gene and stem cell therapy, experimental hematology and molecular biology. In over 200 publications he has contributed to the understanding of stem cells and blood cell development, gene therapy technologies, cancer causation and treatment, human genetic diseases and molecular biology. He serves on Hospital, state and national bodies including Chair of GTTAC, Office of the Gene Technology Regulator – responsible for regulating all genetically-modified organisms in Australia – and immediate past Chair of the Advisory Committee on Biologicals, Therapeutic Goods Administration. Contributions to scientific organizations include co-founding (2000) and past-President (2003–5) of the Australasian Gene & Cell Therapy Society; President (2018–20), President-Elect (2016–18) and Vice President (2008–12) of the International Society for Cell & Gene Therapy; Scientific Advisory Committees and Board member for philanthropic foundations; and several Human Research Ethics Committees. He is a founding Fellow of the Australian Academy of Health and Medical Sciences. In 2018, the Board of the ABC honored him as the sixtieth Boyer Lecturer. He is the recipient of national (RCPA, RACP, ASBMB) and international awards in recognition of his commitment to excellence in medical research, including appointment as an Officer of the Order of Australia.
    15 July 2021
  • Addressing issues in clinical development of AAV-driven gene therapy

    S Sallah
    Sabah Sallah
    Senior Vice President Gene Therapy, Translational Medicine and Hematology at Freeline Therapeutics, Boston, MA, USA
    14 July 2021