CELL & GENE THERAPY INSIGHTS

Scaling up/out: cost-effective and robust transitioning through the clinic to commercial manufacture

Guest Editor:
Jan Thirkettle, Chief Development Officer at Freeline Therapeutics Limited
Jan Thirkettle
Chief Development Officer at Freeline Therapeutics Limited
JAN THIRKETTLE is Chief Development Officer at Freeline, a Syncona-funded start-up focussed on liver-directed AAV gene therapy. Jan has extensive experience in the development and deployment of novel platforms including natural product and enzyme derived NCEs, biologics and gene therapies. Prior to joining Freeline Jan led the establishment of GSK’s Cell & Gene Therapy platform and was responsible for CMC/supply for Strimvelis, the first ex vivo gene therapy to receive an EU Marketing Authorisation Application. He has held industry positions spanning from discovery to commercial manufacturing, but is most passionate about late-state development, new technology introduction and project delivery. Jan holds an MA in Chemistry and a PhD in Biological Chemistry from Oxford University.
September 2020

  • TESSA technology: a new paradigm in AAV manufacturing

    R Cawood
    Ryan Cawood
    CEO at OXGENE
    Ryan founded Oxford Genetics in 2011, after earning a first class degree in genetics and a PhD from Oxford University. The idea behind the company was to simplify and standardise the process of DNA engineering using a proprietary DNA plasmid platform called SnapFast™ that allowed researchers – for the first time – to assemble complex sections of DNA as simply as ‘molecular Lego’. Ryan used his background in genetic engineering and virology to guide and grow the business through a series of strategic changes that explored how further development of the SnapFast™ platform through in house research and development could help overcome multiple challenges in the development of new biologics. This culminated in a rebrand to OXGENE in 2019, as the company redefined itself as a leading solutions provider, using a combination of proprietary technologies to address multiple pinch-points on the journey through design, discovery, development and manufacture of a novel biologic.
    1 October 2020
    FastFacts
  • Scalable and efficient AAV production process with new Fibro chromatography technology

    L Adamson-Small,
    Laura Adamson-Small
    Upstream & Analytical Development Lead, AAV Process Development at UCB Pharma
    Laura Adamson-Small has held multiple roles focused on improving AAV product quality and productivity through optimizing to viral gene expression, delivery, and upstream process design. She currently leads Upstream and Analytical Process Development for AAV vectors at UCB Biosciences in their Early Solutions team. She started her career in AAV supporting multiple aspects of AAV development at the University of Florida Powell Gene Therapy Center. Prior to joining UCB, she previously held led a team focusing on upstream process development and packaging plasmid design improvements for AAV vectors at Homology Medicines.
    M Lundgren,
    Mats Lundgren
    Customer Applications Director at Cytiva
    Mats has more than 25 years of experience in the field of biotechnology. He holds a PhD in Immunology, Cell and Molecular Biology from the Karolinska Institute, Stockholm, Sweden and extensive post-doc training at the MRC Clinical Sciences Centre, Imperial College School of Medicine in London, UK. In his industrial career Mats has had positions as scientist, team manager and Vice President at Pharmacia, AstraZeneca and smaller biotech companies.  In his previous function, Mats was managing both the Cell line and Upstream Process Development teams at a major biotech company.  In his current role, Mats works across different viral vector and vaccine application projects as well as general upstream topics, focusing on customer support, applicability of new technologies and manufacturing solutions.
    P Guterstam
    Peter Guterstam
    Product Manager, Advanced Therapeutics Downstream Solutions at Cytiva
    Peter is Global Product Manager at Cytiva with responsibility for products tailored for downstream processing of Advanced Therapy Medicinal Products. He is based in Uppsala, Sweden. Peter earned is PhD in Neurochemistry from Stockholm University in 2009. He has been with GE since 2003, primarily working in various roles associated with oligonucleotide synthesis and purification. Since GE’s acquisition of Puridify, Peter has the business responsibility to develop products based on the Fibro technology to complement Cytiva’s portfolio of chromatography resins and to develop it further to generate tailored solutions for the manufacture of Advanced Therapy Medicinal Products.
    30 September 2020
    Innovator Insight
  • Delivering cell and gene therapies: evolving logistics considerations - Podcast

    A Lee-Mohan,
    Adrian Lee-Mohan
    Senior Vice President at QuickSTAT
    Adrian Lee-Mohan has been with the Quick Group since 1990, and has held various managementroles in operations, finance and sales. He develops strategic relationships with pharmaceutical andbiotech companies throughout Europe, in order to provide comprehensive logistics solutions for theirglobal supply chain, ensuring product integrity and patient safety. Adrian has extensive experiencein clinical trial logistics, cold chain management and packaging solutions. His focus is on personalizedmedicine, establishing sound supply chain models for Cell/Gene/CAR-T/Immunotherapy, from allphases of clinical trials through to commercialization.
    D Murphy
    David Murphy
    Executive Vice President, Life Sciences at Quick - Specialized Healthcare Logistics
    David Murphy is a 30 year veteran of The Quick Group of Companies, holding various leadership roles in Quick’s Life Science division. Over the past 8 years, David has served as Executive Vice President of Quick Specialized Healthcare Logistics, and works closely with major healthcare organizations to develop specialized logistics solutions to safely transport human organs, tissue, blood and blood products for transplant or research. He also works with biotech and pharmaceutical companies to plan and implement transportation strategies for personalized medicine; including cell, gene and immunotherapy treatments. He develops scalable transportation solutions that preserve the product integrity of these life-saving shipments, and most importantly, the overall safety of patients. He helps to ensure adherence to the strict regulations of the life science industry and the chain of custody at every shipment milestone. David was instrumental in the logistics planning of the first FDA approved cancer vaccine, and subsequent commercialization roll out.
    29 September 2020
    Podcast
  • Scaling up/out: cost-effective and robust transitioning through the clinic to commercial manufacture

    J Thirkettle
    Jan Thirkettle
    Chief Development Officer at Freeline Therapeutics Limited
    JAN THIRKETTLE is Chief Development Officer at Freeline, a Syncona-funded start-up focussed on liver-directed AAV gene therapy. Jan has extensive experience in the development and deployment of novel platforms including natural product and enzyme derived NCEs, biologics and gene therapies. Prior to joining Freeline Jan led the establishment of GSK’s Cell & Gene Therapy platform and was responsible for CMC/supply for Strimvelis, the first ex vivo gene therapy to receive an EU Marketing Authorisation Application. He has held industry positions spanning from discovery to commercial manufacturing, but is most passionate about late-state development, new technology introduction and project delivery. Jan holds an MA in Chemistry and a PhD in Biological Chemistry from Oxford University.
    29 September 2020
    Foreword
  • A necessary transition: why viral vector production for gene therapy needs to evolve

    R Cawood
    Ryan Cawood
    CEO at OXGENE
    Ryan founded Oxford Genetics in 2011, after earning a first class degree in genetics and a PhD from Oxford University. The idea behind the company was to simplify and standardise the process of DNA engineering using a proprietary DNA plasmid platform called SnapFast™ that allowed researchers – for the first time – to assemble complex sections of DNA as simply as ‘molecular Lego’. Ryan used his background in genetic engineering and virology to guide and grow the business through a series of strategic changes that explored how further development of the SnapFast™ platform through in house research and development could help overcome multiple challenges in the development of new biologics. This culminated in a rebrand to OXGENE in 2019, as the company redefined itself as a leading solutions provider, using a combination of proprietary technologies to address multiple pinch-points on the journey through design, discovery, development and manufacture of a novel biologic.
    29 September 2020
    Interview
  • Expert Roundtable: getting a gene therapy product to market: pitfalls and how to prevent them

    H Brahmbhatt,
    Hetal Brahmbhatt
    Principal Investigator at Thermo Fisher Scientific
    Dr. Hetal Brahmbhatt is a Principal Investigator in the Science and Technology Team, Pharma Services, Viral Vector Services at Thermo Fisher Scientific. She has a PhD in Biochemistry and Biomedical Sciences from McMaster University where she characterized small molecule modulators of apoptotic proteins. In her previous role at Thermo Fisher Scientific (former Brammer Bio), she developed and established downstream purification processes for clinical manufacturing of several gene therapy viral vectors. In her present role, she contributes to the development of platform processes and technologies for the manufacturing of viral gene transfer vectors.
    T Guarinoni,
    Thomas Guarinoni
    Downstream Processing at ViralGen Vector Core SL
    Thomas Guarinoni, MSc was recruited in January 2017 as manager of the Downstream Process team at Viralgen. He was successful in transferring the entire purification process from Askbio to Viiralgen in record time. Prior to Viralgen, Thomas Guarinoni worked from 2016 to 2017 at UCB Farchim SA in Switzerland as Process expert supporting the production of CIMZIA® (certolizumab), a monoclonal antibody to neutralize TNF.  He was also involved in the expansion and doubling the production facility. Initially, Thomas Guarinoni started his career at PALL Corporation as technical support engineer (2011-2012), helping the industry to comply with the pharmaceutical regulation linked to every type of filtration method and device. While at Pall Corporation, he switched in 2012 to Bio-purification Process Specialist (2012-2016), assisting Biotechnology companies to develop in-house production process including scaling them up according to process validation, robustness and compliance with GMP. Thomas Guarinoni obtained his MSc in 2011 at ENSTBB (National Superior College of Biotechnology) in Bordeaux, France.
    E Simmons
    Elizabeth Simmons
    Associate Director QC Analytics at Homology Medicines Inc
    29 September 2020
    Expert Roundtable
  • Delivering cell and gene therapies: evolving logistics considerations

    A Lee-Mohan,
    Adrian Lee-Mohan
    Senior Vice President at QuickSTAT
    Adrian Lee-Mohan has been with the Quick Group since 1990, and has held various managementroles in operations, finance and sales. He develops strategic relationships with pharmaceutical andbiotech companies throughout Europe, in order to provide comprehensive logistics solutions for theirglobal supply chain, ensuring product integrity and patient safety. Adrian has extensive experiencein clinical trial logistics, cold chain management and packaging solutions. His focus is on personalizedmedicine, establishing sound supply chain models for Cell/Gene/CAR-T/Immunotherapy, from allphases of clinical trials through to commercialization.
    D Murphy
    David Murphy
    Executive Vice President, Life Sciences at Quick - Specialized Healthcare Logistics
    David Murphy is a 30 year veteran of The Quick Group of Companies, holding various leadership roles in Quick’s Life Science division. Over the past 8 years, David has served as Executive Vice President of Quick Specialized Healthcare Logistics, and works closely with major healthcare organizations to develop specialized logistics solutions to safely transport human organs, tissue, blood and blood products for transplant or research. He also works with biotech and pharmaceutical companies to plan and implement transportation strategies for personalized medicine; including cell, gene and immunotherapy treatments. He develops scalable transportation solutions that preserve the product integrity of these life-saving shipments, and most importantly, the overall safety of patients. He helps to ensure adherence to the strict regulations of the life science industry and the chain of custody at every shipment milestone. David was instrumental in the logistics planning of the first FDA approved cancer vaccine, and subsequent commercialization roll out.
    29 September 2020
    Interview
  • The power of small: success of small academic/healthcare units within the cell & gene therapy industry

    B Weil,
    Benjamin Weil
    Centre for Cell, Gene & Tissue Therapeutics at Royal Free Hospital
    O Bain
    Owen Bain
    Head, GMP ATMP Quality Control, Centre for Cell, Gene & Tissue Therapeutics, Royal Free London NHS Foundation at Royal Free London NHS Foundation Trust
    24 September 2020
    Commentary
  • Driving NK cell therapy manufacture to commercial scale

    L Sender
    Leonard Sender
    Senior Vice President of Medical Affairs for Pediatric, Adolescent and Young Adult Oncology at Nantkwest at NantKwest Inc
    Leonard Sender joined Nantkwest in 2016 as its Senior Vice President of Medical Affairs for Pediatric, Adolescent and Young Adult Oncology. Dr. Sender also serves as Executive Director of the Pediatric, Adolescent and Young Adult Cancer Breakthroughs 2020 Program for the Chan Soon-Shiong Family Foundation. Previously, Dr. Sender served as the Medical Director of the Hyundai Cancer Institute at CHOC (Children’s Hospital, Orange County).  Prior to that, Dr. Sender served as the Medical Director of Clinical Oncology at the University of California’s NCI designated Chao Family Comprehensive Cancer Center.  Dr. Sender collectively brings more than 25 years of experience treating pediatric, adolescent, and young adult and adult cancer patients, and has been the recipient of a $10 mm Hyundai Hope on Wheels grant to study the genomic basis of pediatric and adolescent and young adult cancers to test the hypothesis that genomic knowledge can enable clinical decisions related to treatment options.  In 2010, he founded two entities critical to the development of the emerging adolescent and young adult (AYA) oncology subspecialty as President of the Society for Adolescent and Young Adult Oncology (SAYAO) and Editor-in-Chief of the Journal of Adolescent and Young Adult Oncology (JAYAO). Dr. Sender received his MD from the University of the Witwatersrand in Johannesburg, South Africa and subsequently trained in pediatrics at the University of California, Irvine and in pediatric hematology-oncology at Children’s Hospital Los Angeles.
    23 September 2020
    Interview
  • Expert Roundtable: getting a gene therapy product to market: pitfalls and how to prevent them

    H Brahmbhatt,
    Hetal Brahmbhatt
    Principal Investigator at Thermo Fisher Scientific
    Dr. Hetal Brahmbhatt is a Principal Investigator in the Science and Technology Team, Pharma Services, Viral Vector Services at Thermo Fisher Scientific. She has a PhD in Biochemistry and Biomedical Sciences from McMaster University where she characterized small molecule modulators of apoptotic proteins. In her previous role at Thermo Fisher Scientific (former Brammer Bio), she developed and established downstream purification processes for clinical manufacturing of several gene therapy viral vectors. In her present role, she contributes to the development of platform processes and technologies for the manufacturing of viral gene transfer vectors.
    T Guarinoni,
    Thomas Guarinoni
    Downstream Processing at ViralGen Vector Core SL
    Thomas Guarinoni, MSc was recruited in January 2017 as manager of the Downstream Process team at Viralgen. He was successful in transferring the entire purification process from Askbio to Viiralgen in record time. Prior to Viralgen, Thomas Guarinoni worked from 2016 to 2017 at UCB Farchim SA in Switzerland as Process expert supporting the production of CIMZIA® (certolizumab), a monoclonal antibody to neutralize TNF.  He was also involved in the expansion and doubling the production facility. Initially, Thomas Guarinoni started his career at PALL Corporation as technical support engineer (2011-2012), helping the industry to comply with the pharmaceutical regulation linked to every type of filtration method and device. While at Pall Corporation, he switched in 2012 to Bio-purification Process Specialist (2012-2016), assisting Biotechnology companies to develop in-house production process including scaling them up according to process validation, robustness and compliance with GMP. Thomas Guarinoni obtained his MSc in 2011 at ENSTBB (National Superior College of Biotechnology) in Bordeaux, France.
    E Simmons
    Elizabeth Simmons
    Associate Director QC Analytics at Homology Medicines Inc
    22 September 2020
    Expert Roundtable Video
  • Small labels, big challenges: solutions for advanced therapy labeling

    H Hagen,
    Heidi Hagen
    Chief Strategy Officer at Vineti Inc
    Heidi has been an Operations Executive in the Biotechnology industry for over 25 years and is a Co-founder of Vineti. She has an extensive and proven track record in leading operations and commercializing innovative technologies ranging from recombinant protein/device combinations to the first active immune cell therapy, Provenge. Previously, Heidi was the Global COO for SOTIO, in Prague, Czech Republic with a US office in Boston, MA. Before joining SOTIO she worked for Dendreon for ten years as Senior Vice President of Operations and ten years with Immunex Corporation in a range of roles in drug development and operations management. Heidi has a B.S. in Cell and Molecular Biology, an MS in Bioengineering, and an MBA from the University of Washington.
    C Suchet
    Christophe Suchet
    Chief Product Officer at Vineti Inc
    CHRISTOPHE SUCHET, CHIEF PRODUCT OFFICER, VINETI INC. Christophe brings more than 20 years of information technology and senior biopharmaceutical IT experience to Vineti. Most recently, he served as Vice President of IT for Kite Pharma, Inc., where he developed the essential technology systems that helped Kite’s first cell therapy receive FDA approval, scale rapidly, and secure the landmark sale of the business to Gilead Sciences, Inc. Prior to Kite Pharma, Christophe served as Vice President of IT at Pharmacyclics Inc., a clinical stage and commercial biopharmaceutical company that grew in two years from no product revenue, to $1 billion revenue, to acquisition by AbbVie for $21 billion. Christophe has served as Genentech’s Director, IT Pharma Development Applications, and as IT Director, SAP Center of Excellence & Enterprise Applications. He received his M.S. in Biology and Economy from AgroParisTech.
    21 September 2020
    Commentary
  • Scale-up vs scale-out: evaluating manufacturing scalability of CAR T cell therapy using process simulation tools

    A Katragadda,
    Apoorva Katragadda
    PhD Student at National University of Singapore
    I Karimi,
    Iftekhar Karimi
    Department of Chemical and Biomolecular Engineering at National University of Singapore
    X Wang
    Xiaonan Wang
    Department of Chemical and Biomolecular Engineering at National University of Singapore
    3 September 2020
    Expert Insight
  • The multi-modal manufacturing movement - you don't need to know the future to be ready for it

    The multi-modal manufacturing movement - you don't need to know the future to be ready for it

    Peter Walters
    Peter Walters, ATMP Conceptual Designer at CRB
    Noel Maestre
    Noel Maestre, Director, ATMP at CRB
    30 September 2020
    Webinar
  • Get your assay in gear – removing analytical bottlenecks on the path to clinical readiness

    Get your assay in gear – removing analytical bottlenecks on the path to clinical readiness

    Ian Gaudet
    Ian Gaudet, Senior Director of Process Sciences at Miltenyi Biotec
    8 September 2020
    Webinar
  • Realizing flexible automation – Closing gaps to meet industry needs

    Realizing flexible automation – Closing gaps to meet industry needs

    Knut Niss
    Knut Niss, CTO at Mustang Bio, Inc.
    Steven Thompson
    Steven Thompson, Director, Sales & Product Management at Sexton Biotechnologies
    Mark Lowdell
    Mark Lowdell, Professor of Cell & Tissue Therapy
    12 August 2020
    Webinar
  • Size matters: ensuring donor pool sustainability and cell therapy supply chain success when scaling up and out

    Size matters: ensuring donor pool sustainability and cell therapy supply chain success when scaling up and out

    Chris Learn
    Chris Learn, Senior Director, Therapeutic Science and Strategy Unit at IQVIA
    Amit Agarwal
    Amit Agarwal at Deloitte Consulting
    Raymond Hornung
    Raymond Hornung, Senior Manager, Logistics at Be The Match BioTherapies
    6 August 2020
    Webinar
  • Scalable and efficient AAV production process with new Fibro chromatography technology

    Scalable and efficient AAV production process with new Fibro chromatography technology

    Laura Adamson-Small
    Laura Adamson-Small, Upstream & Analytical Development Lead, AAV Process Development at UCB Pharma
    Mats Lundgren
    Mats Lundgren, Customer Applications Director at Cytiva
    Peter Guterstam
    Peter Guterstam, Product Manager, Advanced Therapeutics Downstream Solutions at Cytiva
    30 July 2020
    Webinar