CELL & GENE THERAPY INSIGHTS

Raw and starting materials

Guest Editor:
Elizabeth Read, Principal Consultant at EJ Read Consulting LLC
Elizabeth Read
Principal Consultant at EJ Read Consulting LLC
Elizabeth Read, MD is an independent consultant focusing on CMC development and CMC regulatory issues for cell- and tissue-based therapies. Dr. Read received her M.D. from the State University of New York (Buffalo, NY). After clinical training in Internal Medicine, Hematology, Oncology, and Blood Banking/Transfusion Medicine, Dr. Read worked at the National Cancer Institute and later in the Clinical Center’s Department of Transfusion Medicine at the National Institutes of Health (NIH; Bethesda, MD), where she served as Section Chief and Medical Director of the Cell Therapy Core Facility from 1995-2006. Initially engaged with novel cellular therapies in the context of hematopoietic transplantation, she later worked on more complex cell, tissue, and gene therapies for a range of clinical indications. From 2007-2010, she headed the Cell Therapy Program at Blood Systems Research Institute (San Francisco, CA), collaborating with UCSF investigators on grant-funded stem cell projects. She previously served as Medical Director at the American Red Cross Blood & Tissue Services (Los Angeles, CA). Over the past 10 years, she held leadership positions at small biotech companies, including Fate Therapeutics (San Diego, CA), StemCyte (Baldwin Park, CA), Medeor Therapeutics (San Mateo, CA), and Adicet Bio (Menlo Park, CA). Dr. Read has authored over 100 scientific publications, and has served as a faculty lecturer at UCSF and for the American Course on Drug Development and Regulatory Science. She has served on advisory committees focused on quality, safety, and efficacy of blood products and cell, tissue, and gene therapies, including the American Association of Blood Banks, the US DHHS/HRSA Advisory Council for Blood Stem Cell Transplantation, and the US Pharmacopeia. She currently serves as an advisor on clinical-stage cell therapy projects funded by the California Institute of Regenerative Medicine.
March 2021

  • Expect the unexpected: maintaining and optimizing a cell collection network during COVID-19

    Expect the unexpected: maintaining and optimizing a cell collection network during COVID-19

    26 March 2021
    Interview
    Amy Hines
    Amy Hines
    Senior Manager, Network Development at Be The Match BioTherapies
    In her role, Amy oversees the performance of apheresis and marrow collection centers and cell therapy labs in the network, and ensures their on-going compliance with FDA and International standards and criteria, industry best practices, and appropriate regulatory and accrediting entities. She joined NMDP/Be The Match in 2013 and managed the NMDP/Be The Match apheresis and marrow collection center network of more than 90 apheresis centers and 80 marrow collection centers. Amy has nearly 20 years of experience in the cellular therapy field. Her career in this field began as a stem cell transplant registered nurse at Spectrum Health/Helen DeVos Children’s Hospital in Grand Rapids, Mich., in 1999. Over the course of 12 years in Grand Rapids, Amy held a wide range of positions including Staff Nurse on a 15-bed pediatric unit, Transplant Coordinator, Program Manager, Clinical Research Coordinator of the Children’s Oncology Group (COG), and finally Program Administrator for the pediatric and adult stem cell transplantation programs.
  • Managing raw and starting materials for viral vector manufacture: managing risk and staying flexible

    Managing raw and starting materials for viral vector manufacture: managing risk and staying flexible

    26 March 2021
    Interview
    Eric Hacherl,
    Eric Hacherl
    Head of Manufacturing at Spark Therapeutics, Inc
    Christopher Klem,
    Christopher Klem
    External Manufacturing and CDMO Lead at Spark Therapeutics, Inc
    Mark Galbraith,
    Mark Galbraith
    Head of Quality Control & Analytical Sciences at Spark Therapeutics, Inc
    Ryan Bartock
    Ryan Bartock
    Vice President and Head,Technical Strategy & Operations at Spark Therapeutics, Inc
  • Expect the unexpected: maintaining and optimizing a cell collection network during COVID-19

    A Hines
    Amy Hines
    Senior Manager, Network Development at Be The Match BioTherapies
    In her role, Amy oversees the performance of apheresis and marrow collection centers and cell therapy labs in the network, and ensures their on-going compliance with FDA and International standards and criteria, industry best practices, and appropriate regulatory and accrediting entities. She joined NMDP/Be The Match in 2013 and managed the NMDP/Be The Match apheresis and marrow collection center network of more than 90 apheresis centers and 80 marrow collection centers. Amy has nearly 20 years of experience in the cellular therapy field. Her career in this field began as a stem cell transplant registered nurse at Spectrum Health/Helen DeVos Children’s Hospital in Grand Rapids, Mich., in 1999. Over the course of 12 years in Grand Rapids, Amy held a wide range of positions including Staff Nurse on a 15-bed pediatric unit, Transplant Coordinator, Program Manager, Clinical Research Coordinator of the Children’s Oncology Group (COG), and finally Program Administrator for the pediatric and adult stem cell transplantation programs.
    26 March 2021
    Interview
  • Managing raw and starting materials for viral vector manufacture: managing risk and staying flexible

    E Hacherl,
    Eric Hacherl
    Head of Manufacturing at Spark Therapeutics, Inc
    C Klem,
    Christopher Klem
    External Manufacturing and CDMO Lead at Spark Therapeutics, Inc
    M Galbraith et al.
    Mark Galbraith
    Head of Quality Control & Analytical Sciences at Spark Therapeutics, Inc
    26 March 2021
    Interview
  • Key considerations for hMSC product and process development through clinical cGMP manufacturing

    Key considerations for hMSC product and process development through clinical cGMP manufacturing

    Jon Rowley
    Jon Rowley, Founder and CPO, RoosterBio Inc.
    Rita Barcia
    Rita Barcia, Vice President of Research and Development, Sentien Biotech
    Joe Candiello
    Joe Candiello, Senior Product Manager, RoosterBio Inc.
    Joseph Takacs
    Joseph Takacs, Field Application Scientist, RoosterBio Inc.
    Jan Nolta
    Jan Nolta, Director - Stem Cell Program Sacramento at University of California Davis
    Carolyn Yeago
    Carolyn Yeago, Associate Director at Georgia Institute of Technology
    Jim Birkhead
    Jim Birkhead, Director, Research & Development at Vericel Corporation
    28 April 2021
    Webinar
  • Raw and starting materials

    E Read
    Elizabeth Read
    Principal Consultant at EJ Read Consulting LLC
    Elizabeth Read, MD is an independent consultant focusing on CMC development and CMC regulatory issues for cell- and tissue-based therapies. Dr. Read received her M.D. from the State University of New York (Buffalo, NY). After clinical training in Internal Medicine, Hematology, Oncology, and Blood Banking/Transfusion Medicine, Dr. Read worked at the National Cancer Institute and later in the Clinical Center’s Department of Transfusion Medicine at the National Institutes of Health (NIH; Bethesda, MD), where she served as Section Chief and Medical Director of the Cell Therapy Core Facility from 1995-2006. Initially engaged with novel cellular therapies in the context of hematopoietic transplantation, she later worked on more complex cell, tissue, and gene therapies for a range of clinical indications. From 2007-2010, she headed the Cell Therapy Program at Blood Systems Research Institute (San Francisco, CA), collaborating with UCSF investigators on grant-funded stem cell projects. She previously served as Medical Director at the American Red Cross Blood & Tissue Services (Los Angeles, CA). Over the past 10 years, she held leadership positions at small biotech companies, including Fate Therapeutics (San Diego, CA), StemCyte (Baldwin Park, CA), Medeor Therapeutics (San Mateo, CA), and Adicet Bio (Menlo Park, CA). Dr. Read has authored over 100 scientific publications, and has served as a faculty lecturer at UCSF and for the American Course on Drug Development and Regulatory Science. She has served on advisory committees focused on quality, safety, and efficacy of blood products and cell, tissue, and gene therapies, including the American Association of Blood Banks, the US DHHS/HRSA Advisory Council for Blood Stem Cell Transplantation, and the US Pharmacopeia. She currently serves as an advisor on clinical-stage cell therapy projects funded by the California Institute of Regenerative Medicine.
    26 March 2021
    Foreword
  • The importance of starting materials: quality and regulatory considerations for cell-based therapies

    B Bonamassa,
    Barbara Bonamassa
    Quality Assessor, ATMPs at AIFA (Italian Medicines Agency)
    P Gasparini,
    Paolo Gasparini
    Head, Department of Advanced Diagnostics & Clinical Research, Institute for Maternal & Child Health - IRCCS Burlo Garofolo at University of Trieste
    G Pompilio et al.
    Giulio Pompilio
    Professor at Università degli Studi di Milano
    25 March 2021
    Regulatory Insight
  • Developing an understanding of the analytical landscape for testing complex biological raw materials in advanced therapy medicinal products: a CRO perspective

    A Michel,
    Alistair Michel
    Senior Scientist I, Biomolecular Analysis at RSSL
    Alistair is an immunologist and has a BSc (Hons) from the University of Edinburgh and an MSc from Imperial College London. He is a member of the British Society of Immunologists. He has over 15 years’ experience developing immunoassays, ten of which have been spent developing and validating/verifying ELISAs (both sandwich & competitive) to ICH and ISO13485 standards. He has experience of Western Blot, T-Cell proliferation assays, SDS PAGE, Molecular Biology (DNA/RNA extraction from whole blood and performance of assays utilising PCR & RT-PCR), Bacterial and Mammalian Cell Culture, Microscopy & Histology. He has extensive project management experience, which includes planning, co-ordination with clients, organisation and execution of work to meet set timeframes.
    D Neville
    David Neville
    Senior Associate Principal Scientist, Biomolecular Analysis at RSSL
    25 March 2021
    Innovator Insight
  • The case for the use of pooled donors in the manufacture of allogeneic cell therapies

    B Weil,
    Benjamin Weil
    Head, GMP Engineering at Royal Free London NHS Foundation Trust
    M Lowdell
    Mark Lowdell
    Professor of Cell & Tissue Therapy at Royal Free London NHS Foundation Trust
    Mark trained as an immunopathologist at the Royal London Hospital and moved to the Royal Free Hospital/UCL in 1994 to set up the immunotherapy programme in malignant hematology. He has specialist knowledge of cellular therapeutics and is the UK representative on the Joint Accreditation Committee ISCT/EBMT (JACIE), which is responsible for setting and co-ordinating standards for cellular therapies across Europe. Mark holds Qualified Person status for the release of investigational somatic cell therapy medicinal products in the EU and is a Designated Individual under a Human Tissue Authority licence for therapeutic cells. Mark has been a Chief Scientific Officer and Chief Manufacturing Officer since the formation of the InMuneBio in September 2015. Since February 2009, he has also been Director of Cellular Therapy at the Royal Free London NHS Foundation Trust. He received his PhD in clinical immunology from London Hospital Medical College, University of London in 1992 and is a qualified immunopathologist.
    24 March 2021
    Expert Insight
  • Master ATMP processes by controlling raw and starting material

    A Lebrun,
    Anne-Sophie Lebrun
    Production & Optimisation Manager at Bone Therapeutics SA
    C Brenner
    Carmen Brenner
    Associate Director, QA at Bone Therapeutics SA
    24 March 2021
    Commentary
  • Fitting product to process: raw materials customization for cell therapy manufacturing success

    L Belcastro,
    Lili Belcastro
    Scientist, Biotherapeutics Material Sciences at Janssen
    Lili Belcastro is a Scientist at Janssen and is responsible for the qualification of raw materials used in Janssen’s cell and gene therapy products. Dr Belcastro has over 10 years of experience in preclinical and clinical cancer biology, cell and gene therapy product development, and analytical method development. Prior to joining Janssen in 2017, she was finishing her PhD in cancer biology in a joint program with the University of the Sciences and The Wistar Institute in Philadelphia. Prior to joining industry, Dr. Belcastro led pediatric, preclinical, in vivo testing programs at the Children’s Hospital of Philadelphia for small molecule inhibitors, oncolytic viruses, antibody-drug conjugates, and radiopharmaceuticals. Dr. Belcastro holds two bachelor’s degrees from Temple University and the University of the Sciences.
    M Brabec,
    Mitchell Brabec
    Commercial Development Specialist at Bio-techne
    Since 2015, Mitch has filled multiple roles within Bio-Techne prior to joining the Commercial Development group. He began in Technical Support to support Research Use Only (RUO) and Good Manufacturing Practices (GMP) products for academic and industrial clients, followed by Product Marketing in the Protein Business Unit for RUO and GMP proteins. As a Commercial Development Specialist with the Custom Projects team, he helps clients find solutions for their workflows by managing GMP conversion, custom protein modifications and GMP fill-finish projects. Mitch works closely with Bio-Techne’s development scientists and expert quality and regulatory groups.
    L Clarke et al.
    Lindsey Clarke
    Global Product Marketing & Commercial Strategy, Cell & Gene Therapy at Bio-techne
    Lindsey has spent the last decade working in Cell and Gene Therapy on the raw materials supplier side prior to running Bio-Techne’s Global Cell and Gene Therapy Product Marketing team. She joined Bio-Techne in 2018 to establish their European Cell and Gene Therapy specialist team and has recently transitioned into a global role. She is focused on building the team to support customers applications, planning the roll out of new innovations, and developing strategic partnerships within the industry. Prior to this she was in Miltenyi Biotec’s UK field team, working closely with numerous process development and manufacturing teams to assist in translating their varied cell therapies to the clinic. Lindsey holds a first-class degree in Pharmacology from the University of Bath, a PhD in Immunology from UCL and worked as a postdoctoral scientist at Imperial College before moving into more commercial roles.
    22 March 2021
    Innovator Insight
  • Emerging trends in control and regulation of immunotherapy cellular starting materials

    N Manley
    Nate Manley
    Senior Consultant, Dark Horse Consulting
    Nathan Manley, PhD is a senior consultant at Dark Horse Consulting with over 17 years of experience in stem cell biology, neuroscience, and preclinical modeling of human disease. Prior to joining Dark Horse, he led research and preclinical development efforts at Asterias Biotherapeutics, a pluripotent stem cell-based biotech company, and trained as an academic researcher in the departments of Biology and Neurosurgery at Stanford University. Over the course of his career, Manley has contributed to the advancement of a wide range of experimental and clinical-stage cell therapy products, authored scientific articles, and co-authored cell therapy patent applications. He also is a part-time instructor in the Biology Department at San Jose State University.
    19 March 2021
    Interview
  • Considerations for performing virtual quality audits on manufacturers of gene therapy viral vectors: an auditor’s perspective during the COVID-19 public health emergency

    G du Moulin
    Gary C du Moulin, PhD, MPH, RAC
    Independent consultant (Fairlead Quality Compliance) Former, V.P., Quality Operations, Genzyme Biosurgery
    18 March 2021
    Commentary
  • Sourcing and procurement best practices in the face of uncertainty

    R Stout
    Richard Stout
    Adaptimmune Therapeutics
    Richard Stout has 20 years of Sourcing, Procurement, and External Manufacturing experience. Having spent his early career in the electronics and elevator manufacturing fields with Schindler Elevator and Tyco Electronics, he gained big pharma experience working in the vaccines and biologics divisions of both GSK and Merck, before bringing his expertise to the cell and gene therapy space with Adaptimmune. He has an MBA from Mt. St. Mary’s University, Emmitsburg, MD.
    18 March 2021
    Interview
  • Considerations for controlling the variability of raw biological materials in the manufacturing of autologous patient-specific therapies

    S Zvonic
    Sanjin Zvonic
    Established leader in development and manufacturing of cell-based therapeutics. Cell and molecular biologist with a strong technical/scientific background in physiology, stem cell biology, and cell therapy CMC development. After earning his Doctorate in Cell and Molecular Biology Dr. Zvonić continued on to a post-doctoral position at the Pennington Biomedical Research Center where he contributed to the development of methodologies for the isolation and culture of human adipose stem cells (ASC) from several sources, and later utilized ASCs to develop a human in vitro model for circadian biology studies. He then transitioned onto a second post-doctoral role at Tulane University, where he directed the technology transfer and development of bone marrow-derived MSC manufacturing and analytical methods in the in-house GMP production facility.In 2009, Dr. Zvonić joined PCT, where he focused on client engagement and technology transfer into PCT, giving him a comprehensive understanding of cell therapy development, manufacturing and commercialization requirements and strategies. In 2014, he joined Novartis Cell and Gene Therapy Unit, where he focused on the development and commercialization of Novartis C/GT pipeline products. In 2016 Dr. Zvonić returned to PCT, with a focus on driving the growth and development of PCT’s clinical and commercial manufacturing business lines while integrating into Hitachi Chemical.In his current role at WindMIL Therapeutics, Dr. Zvonić leads the development of WindMIL’s core technologies and pipeline products, while concurrently contributing to the organizational growth and development.
    16 March 2021
    Editorial
  • Upholding raw material suppliers to higher quality standards to better support cell and gene therapy manufacturers

    M Nelson
    Monica C. Nelson, PhD
    Director, Quality Control, Century Therapeutics
    5 March 2021
    Expert Insight
  • Fitting product to process: raw materials customization for cell therapy manufacturing success

    Fitting product to process: raw materials customization for cell therapy manufacturing success

    John Paul Tomtishen III
    John Paul Tomtishen III, Director of Manufacturing, Technical Operations at Legend Biotech Co.
    Lindsey Clarke, PhD
    Lindsey Clarke, PhD , Global Product Marketing & Commercial Strategy, Cell & Gene Therapy, Bio-Techne
    Lili Belcastro, PhD
    Lili Belcastro, PhD, Scientist, Biotherapeutics Material Sciences,Janssen Pharmaceutical R&D
    Raymond Luke
    Raymond Luke, Associate Director, MS&T, Adaptimmune
    Mitchell Brabec
    Mitchell Brabec, Commercial Development Specialist, Bio-techne
    11 February 2021
    Webinar
  • Focusing on donor characteristics and efficient selection to improve allogeneic cell therapy outcomes

    Focusing on donor characteristics and efficient selection to improve allogeneic cell therapy outcomes

    Martin Maiers, MS
    Martin Maiers, MS, Vice President of Biomedical Informatics,  Center for Blood and Marrow Transplant Research (CIBMTR)
    Blake Aftab, PhD
    Blake Aftab, PhD, Vice President, Preclinical & Translational Science, Atara Biotherapeutics, Inc.
    Daniel Gibson
    Daniel Gibson, Director of Cell and Gene Therapy Services, Anthony Nolan
    Joanne Kurtzberg, PhD
    Joanne Kurtzberg, PhD, Director, Carolinas Cord Blood Bank, Duke University
    Dorit Harati
    Dorit Harati, Former Vice President, Quality Assurance, Manufacturing and Logistics, Gamida Cell
    21 January 2021
    Webinar
  • Raw materials of biological origin: navigating regulatory requirements for cell therapies

    Raw materials of biological origin: navigating regulatory requirements for cell therapies

    Marlin Frechette
    Marlin Frechette, Chief Quality & Compliance Officer, FUJIFilm Irvine Scientific
    Dominic Clarke
    Dominic Clarke, Global Head of Cell Therapy, HemaCare Corporation
    13 January 2021
    Webinar