CELL & GENE THERAPY INSIGHTS

Clinical trial design, supply chain and operations

July 2021

  • Preparing for pivotal: solving challenges in scale for cell and gene therapy clinical trials

    Preparing for pivotal: solving challenges in scale for cell and gene therapy clinical trials

    6 August 2021
    Innovator Insight
    Subbu Viswanathan,
    Subbu Viswanathan
    Compliance Officer and SVP of Quality, Security, and Compliance, Vineti
    Richard Gaeto,
    Richard Gaeto
    Senior Vice President, Technical Operations at Iovance Biotherapeutics
    Rich has been a Technical Operations Executive in the biotech and the Cell and Gene therapy industry for over 30 years. He has an extensive background in commercial launches such as Enbrel, Provenge, Neulasta, and Recothrom, global supply chain strategies, and systems integration regarding personalized medicine, Chain of Custody and Chain of Identity. He has held executive positions at Iovance as Sr. Vice President of Technical Operations, Vice President of Technical Operations at ZymoGenetics and Vice President of Commercial Operations at Dendreon. In addition to on-going consulting roles, Rich has recently completed a full-time Head of Operations position at Imvax focusing on CDMO selection, build out of internal manufacturing, and establishment of an integrated solution regarding CRO, Manufacturing, and Patient Scheduling. Prior to these activities Rich has held positions at Amgen, Immunex, and Centocor.
    Erin Goodhue Meyer,
    Erin Goodhue Meyer
    Attending Physician, Transfusion Service and Apheresis Clinic, Nationwide Children’s Hospital and Director of Clinical Research and Development at Terumo BCT
    Chris Greenberg,
    Chris Greenberg
    Associate Director, Clinical CAR-T Systems at Janssen Pharmaceutical companies of Johnson & Johnson
    Jim Wise
    Jim Wise
    Vice President, Head of Center for Immuno-Oncology, Cellular & Gene Therapy at PRA Health Sciences
    Jim has long-term experience managing early through late clinical development, with a primary focus on hematology/oncology indications and advanced therapeutics. Currently, he heads PRA’s Center for Immuno-Oncology, Cellular & Gene Therapy which centralizes cross-functional knowledge expertise and global capabilities in these treatment modalities.
  • Preparing for pivotal: solving challenges in scale for cell and gene therapy clinical trials

    S Viswanathan,
    Subbu Viswanathan
    Compliance Officer and SVP of Quality, Security, and Compliance, Vineti
    R Gaeto,
    Richard Gaeto
    Senior Vice President, Technical Operations at Iovance Biotherapeutics
    Rich has been a Technical Operations Executive in the biotech and the Cell and Gene therapy industry for over 30 years. He has an extensive background in commercial launches such as Enbrel, Provenge, Neulasta, and Recothrom, global supply chain strategies, and systems integration regarding personalized medicine, Chain of Custody and Chain of Identity. He has held executive positions at Iovance as Sr. Vice President of Technical Operations, Vice President of Technical Operations at ZymoGenetics and Vice President of Commercial Operations at Dendreon. In addition to on-going consulting roles, Rich has recently completed a full-time Head of Operations position at Imvax focusing on CDMO selection, build out of internal manufacturing, and establishment of an integrated solution regarding CRO, Manufacturing, and Patient Scheduling. Prior to these activities Rich has held positions at Amgen, Immunex, and Centocor.
    E Goodhue Meyer et al.
    Erin Goodhue Meyer
    Attending Physician, Transfusion Service and Apheresis Clinic, Nationwide Children’s Hospital and Director of Clinical Research and Development at Terumo BCT
    6 August 2021
    Innovator Insight
  • CRISPR gene editing for sickle cell disease: one disease, differentiated approaches

    L Michaels,
    Lisa A Michaels, MD
    Chief Medical Officer, Editas Medicine
    B Eaton
    Bruce E Eaton, PhD
    Chief Business Officer, Editas Medicine
    5 August 2021
    Editorial
  • Strengthening the clinical supply chain for individualized therapies

    A Case
    James Andrew Case
    Head of Clinical Supply Chain – Individualized Therapies, Genentech
    5 August 2021
    Interview
  • Driving clinical progress of gene therapy in cystic fibrosis

    U Griesenbach
    Uta Griesenbach
    NHLI, Imperial College, Manresa Rd london SW3 6LR
    28 July 2021
    Interview
  • 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
    Interview
  • 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
    Interview
  • 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
    Interview