CELL & GENE THERAPY INSIGHTS

Cell therapy CMC and quality control

Guest Editor:
Christiane Niederlaender, Vice President Technical at Parexel
Christiane Niederlaender
Vice President Technical at Parexel
Christiane Niederlaender. has spent 9 years at the UK medicines regulator MHRA, most recently as the Unit Manager of the Biologicals Unit. In that capacity she chaired and oversaw all formal scientific advice meetings and had in-depth involvement of Brexit preparedness. At the same time Christiane was a Senior Assessor and the UK delegate at the EMA committee for advanced therapies (CAT). She was UK CAT Rapporteur responsible for managing the portfolio of all UK ATMP products (cell and gene therapies). She was also the Rapporteur for the EMA CAT Gene Therapy Guideline and quality lead for gene therapy section of new draft guideline for investigational ATMPs. She was a drafting group member in all other quality related ATMP drafting groups, including GMP for ATMPs. Prior to joining MHRA, Christiane worked with the UK regulator for the European Tissues and Cells Directive as a quality manager and inspector. Christiane has experience in CMC and regulatory strategy for all types of biological products, with a particular focus on gene and cell therapies, novel biotherapeutics, biosimilars and vectored vaccines. While at MHRA, she was the principal quality/CMC assessor for 10 centralized MAAs, including two full dossier applications for ATMPs as sole UK quality assessor (including CAR-T, see publications). She has in depth knowledge of all European procedure types, both centralized and MR/DCP, and can give advice on ATMP classification, ODD and the PRIME program. As an ex-member of the EMA CAT committee, Christiane is familiar with the thinking of all European regulators in the ATMP space and is used to dialogue and negotiation with many regulatory agencies, as well as presenting her thinking during committee discussions. Christiane undertook her undergraduate studies in biochemistry, neurobiology and molecular biology at Heidelberg University in Germany before obtaining her PhD in molecular developmental neurobiology at Kings College London in the UK. Christiane has published her academic research in many high impact journals, including Nature.
May 2021

  • Cell therapy CMC and quality control

    Cell therapy CMC and quality control

    14 May 2021
    Foreword
    Christiane Niederlaender
    Christiane Niederlaender
    Vice President Technical at Parexel
    Christiane Niederlaender. has spent 9 years at the UK medicines regulator MHRA, most recently as the Unit Manager of the Biologicals Unit. In that capacity she chaired and oversaw all formal scientific advice meetings and had in-depth involvement of Brexit preparedness. At the same time Christiane was a Senior Assessor and the UK delegate at the EMA committee for advanced therapies (CAT). She was UK CAT Rapporteur responsible for managing the portfolio of all UK ATMP products (cell and gene therapies). She was also the Rapporteur for the EMA CAT Gene Therapy Guideline and quality lead for gene therapy section of new draft guideline for investigational ATMPs. She was a drafting group member in all other quality related ATMP drafting groups, including GMP for ATMPs. Prior to joining MHRA, Christiane worked with the UK regulator for the European Tissues and Cells Directive as a quality manager and inspector. Christiane has experience in CMC and regulatory strategy for all types of biological products, with a particular focus on gene and cell therapies, novel biotherapeutics, biosimilars and vectored vaccines. While at MHRA, she was the principal quality/CMC assessor for 10 centralized MAAs, including two full dossier applications for ATMPs as sole UK quality assessor (including CAR-T, see publications). She has in depth knowledge of all European procedure types, both centralized and MR/DCP, and can give advice on ATMP classification, ODD and the PRIME program. As an ex-member of the EMA CAT committee, Christiane is familiar with the thinking of all European regulators in the ATMP space and is used to dialogue and negotiation with many regulatory agencies, as well as presenting her thinking during committee discussions. Christiane undertook her undergraduate studies in biochemistry, neurobiology and molecular biology at Heidelberg University in Germany before obtaining her PhD in molecular developmental neurobiology at Kings College London in the UK. Christiane has published her academic research in many high impact journals, including Nature.
  • The role of big data analytics in digitized manufacturing

    D Marshall,
    Damian Marshall
    Cell and Gene Therapy Catapult, London, UK
    Dr DamiAn Marshall is the director of new and enabling technologies at the Cell and Gene Therapy Catapult and has almost 20 years of industrial experience gained working for SME’s and large companies. He is responsible for providing vision, expertise and leadership to a team of ~70 scientists working with a wide range of cell and gene therapy developers. Together they are addressing some of the barriers to growth within the industry, supporting the development of new technologies and helping create an advantageous environment that gives companies working in the UK a sustained competitive advantage.
    J Churchwell,
    John Churchwell
    Cell and Gene Therapy Catapult, London, UK
    M Baradez
    Marc-Olivier Baradez
    Cell and Gene Therapy Catapult, London, UK
    Marc-Olivier Baradez is Lead Scientist in the Industrialisation team at the Cell and Gene Therapy Catapult. He has over 13 years of industrial experience in the implementation of analytical strategies for the characterisation of cell therapies. He is currently the Catapult’s Strategic Lead for its core Data programme, focusing on high-throughput data generation, data handling and analysis, innovative methodologies for Quality-by-Design and in-process controls in the context of Intelligent Manufacturing, across a wide portfolio of cell and gene therapy products and processes.Marc-Olivier’s multidisciplinary expertise lies in experimental strategies supporting high-throughput multivariate quantitative approaches, in which established and new technologies are integrated to characterise cell products and their manufacturing processes. He was previously Science Leader in Cell Metrology at LGC, the UK designated National Measurement Institute for chemical and bio-measurements. He started his industrial career as Senior Scientist at ReNeuron, where he characterised the company’s first clinical neural stem cell line for stroke therapy.
    14 May 2021
    Expert Insight
  • Outcomes from a cell viability workshop: fit-for-purpose considerations for cell viability measurements for cellular therapeutic products

    L Pierce,
    Laura Pierce
    Author for correspondence: Biosystems and Biomaterials Division, Materials Measurement Laboratory, National Institute of Standards and Technology, Gaithersburg, MD, USA laura.pierce@nist.gov
    S Sarkar,
    Sumona Sarkar
    Biosystems and Biomaterials Division, Materials Measurement Laboratory, National Institute of Standards and Technology, Gaithersburg, MD, USA
    L Chan et al.
    Leo Li-Ying Chan
    Department of Advanced Technology R&D, Nexcelom Bioscience LLC, Lawrence, MA, USA
    14 May 2021
    Commentary
  • Bioprocess and analytics priorities for the tissue engineered product and cell therapy fields

    J Allickson
    Julie Allickson, PhD
    Chief Manufacturing Development Center Officer Wake Forest Institute for Regenerative Medicine, Wake Forest School of Medicine
    14 May 2021
    Interview
  • Addressing sampling challenges in early phase cell therapy manufacturing

    S Mgebroff
    Stephanie Mgebroff
    Therapeutic Cell Production Core (TCPC), Seattle Children’s Therapeutics (SCTx), Seattle Children’s, Seattle WA, USA
    14 May 2021
    Expert Insight
  • Unleashing the full power of next gen bioanalytics on cell therapy development

    E Alonzo
    Eric Alonzo, PhD
    Strategy Lead – Oncology, Analytical Development, CMC Lead, bbT369 Program, bluebird bio
    14 May 2021
    Interview
  • Finding quality in complexity: how cellular therapeutics are shifting analytical paradigms for clinical supply and product manufacturing

    V Jimenez Sabinina,
    Vilma Jimenez Sabinina
    Analytical Development and Clinical QC for Biotech Products, Bayer AG, Product Supply, Friedrich- Ebert-Straße 217-233, Bldg. 230, Room 2.205, 42117 Wuppertal, Germany
    D Hildebrand
    Dominic Günter Hildebrand
    Author for correspondence: Bayer AG, Product Supply, Analytical Development and Clinical QC for Biotech Products, Friedrich- Ebert-Straße 217-233, Bldg. 230, Room 2.235, 42117 Wuppertal, Germany dominic.hildebrand@bayer.com
    14 May 2021
    Commentary
  • Characterization of a novel high-throughput, high-speed and high-precision plate-based image cytometric cell counting method

    J Bell,
    Jordan Bell
    Department of Advanced Technology R&D, Nexcelom Bioscience LLC, Lawrence, MA 01843, USA
    Y Huang,
    Yongyang Huang
    Department of Advanced Technology R&D, Nexcelom Bioscience LLC, Lawrence, MA 01843, USA
    H Qazi et al.
    Henry Qazi
    Department of Advanced Technology R&D, Nexcelom Bioscience LLC, Lawrence, MA 01843, USA
    14 May 2021
    Research Article
  • Cell therapy CMC and quality control

    C Niederlaender
    Christiane Niederlaender
    Vice President Technical at Parexel
    Christiane Niederlaender. has spent 9 years at the UK medicines regulator MHRA, most recently as the Unit Manager of the Biologicals Unit. In that capacity she chaired and oversaw all formal scientific advice meetings and had in-depth involvement of Brexit preparedness. At the same time Christiane was a Senior Assessor and the UK delegate at the EMA committee for advanced therapies (CAT). She was UK CAT Rapporteur responsible for managing the portfolio of all UK ATMP products (cell and gene therapies). She was also the Rapporteur for the EMA CAT Gene Therapy Guideline and quality lead for gene therapy section of new draft guideline for investigational ATMPs. She was a drafting group member in all other quality related ATMP drafting groups, including GMP for ATMPs. Prior to joining MHRA, Christiane worked with the UK regulator for the European Tissues and Cells Directive as a quality manager and inspector. Christiane has experience in CMC and regulatory strategy for all types of biological products, with a particular focus on gene and cell therapies, novel biotherapeutics, biosimilars and vectored vaccines. While at MHRA, she was the principal quality/CMC assessor for 10 centralized MAAs, including two full dossier applications for ATMPs as sole UK quality assessor (including CAR-T, see publications). She has in depth knowledge of all European procedure types, both centralized and MR/DCP, and can give advice on ATMP classification, ODD and the PRIME program. As an ex-member of the EMA CAT committee, Christiane is familiar with the thinking of all European regulators in the ATMP space and is used to dialogue and negotiation with many regulatory agencies, as well as presenting her thinking during committee discussions. Christiane undertook her undergraduate studies in biochemistry, neurobiology and molecular biology at Heidelberg University in Germany before obtaining her PhD in molecular developmental neurobiology at Kings College London in the UK. Christiane has published her academic research in many high impact journals, including Nature.
    14 May 2021
    Foreword