CELL & GENE THERAPY INSIGHTS

Vol 7 Issue 4

Apr 2021

FEATURING

Issue Spotlight: Cell therapy CMC and quality control

Latest articles

Supply Chain Focus: Ensuring supply chain scalability for cell & gene therapy products

Report: Clinical Trends

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Spotlight

Cell therapy CMC and quality control

May 2021
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.

Foreword

Cell therapy CMC and quality control

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.
14 May 2021
Foreword

Research Article

Characterization of a novel high-throughput, high-speed and high-precision plate-based image cytometric cell counting method

Jordan Bell,
Jordan Bell
Department of Advanced Technology R&D, Nexcelom Bioscience LLC, Lawrence, MA 01843, USA
Yongyang Huang,
Yongyang Huang
Department of Advanced Technology R&D, Nexcelom Bioscience LLC, Lawrence, MA 01843, USA
Henry Qazi,
Henry Qazi
Department of Advanced Technology R&D, Nexcelom Bioscience LLC, Lawrence, MA 01843, USA
Dmitry Kuksin,
Dmitry Kuksin
Department of Advanced Technology R&D, Nexcelom Bioscience LLC, Lawrence, MA 01843, USA
Jean Qiu,
Jean Qiu
Department of Advanced Technology R&D, Nexcelom Bioscience LLC, Lawrence, MA 01843, USA
Bo Lin,
Bo Lin
Department of Advanced Technology R&D, Nexcelom Bioscience LLC, Lawrence, MA 01843, USA
Leo Li-Ying Chan
Leo Li-Ying Chan
Author for correspondence Department of Advanced Technology R&D, Nexcelom Bioscience LLC, Lawrence, MA 01843, USA lchan@nexcelom.com
14 May 2021
Research Article

Expert Insight

The role of big data analytics in digitized manufacturing

Damian 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.
John Churchwell,
John Churchwell
Cell and Gene Therapy Catapult, London, UK
Marc-Olivier 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

Addressing sampling challenges in early phase cell therapy manufacturing

Stephanie Mgebroff
Stephanie Mgebroff
Therapeutic Cell Production Core (TCPC), Seattle Children’s Therapeutics (SCTx), Seattle Children’s, Seattle WA, USA
14 May 2021
Expert Insight

Commentary

Outcomes from a cell viability workshop: fit-for-purpose considerations for cell viability measurements for cellular therapeutic products

Laura 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
Sumona Sarkar,
Sumona Sarkar
Biosystems and Biomaterials Division, Materials Measurement Laboratory, National Institute of Standards and Technology, Gaithersburg, MD, USA
Leo Li-Ying Chan,
Leo Li-Ying Chan
Department of Advanced Technology R&D, Nexcelom Bioscience LLC, Lawrence, MA, USA
Bo Lin,
Bo Lin
Department of Advanced Technology R&D, Nexcelom Bioscience LLC, Lawrence, MA, USA
Jean Qiu
Jean Qiu
Department of Advanced Technology R&D, Nexcelom Bioscience LLC, Lawrence, MA, USA
14 May 2021
Commentary

Finding quality in complexity: how cellular therapeutics are shifting analytical paradigms for clinical supply and product manufacturing

Vilma 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
Dominic Günter 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

Interview

Bioprocess and analytics priorities for the tissue engineered product and cell therapy fields

Julie Allickson, PhD
Julie Allickson, PhD
Chief Manufacturing Development Center Officer Wake Forest Institute for Regenerative Medicine, Wake Forest School of Medicine
14 May 2021
Interview

Unleashing the full power of next gen bioanalytics on cell therapy development

Eric Alonzo, PhD
Eric Alonzo, PhD
Strategy Lead – Oncology, Analytical Development, CMC Lead, bbT369 Program, bluebird bio
14 May 2021
Interview

Supply Chain FOCUS

Interview

Building clinical and commercial allogeneic CAR T cell therapy supply chains

Bob Amareld
Bob Amareld
Head of Supply Chain, Precision Biosciences
Bob Amareld is the Head of Supply Chain at Precision Biosciences, leading the foundational build out of our Cell and Gene Therapy Supply Chain from the ground up, including our Plan-Source-Make-Deliver functions. Following our establishment of our baseline functions, we have begun adding SAAS technology suites for sourcing and inventory, as well as phase appropriate logistics, inventory management, sourcing, and analytics dashboarding to support our clinical trials and internal allogeneic CAR-T and gene vector manufacturing suites. Previously Bob came from Big Biotech at Biogen where he led the Strategic Operations and Technology function and headed the digital transformation of Strategic Sourcing by bringing in technology such as automated spend analytics, E-sourcing, advanced data analysis, market intelligence and Contract Lifecycle Management along with forming distinct Global Centers of Excellence which included a BPO Buying Desk , Contract Life Cycle COE and Strategic Sourcing COE, enabling savings totaling over $500MM in just 3 years. Additionally, Bob has diverse experience in Supply Chain, Procurement, Sourcing, Biomedical and Process Engineering, Intellectual Property Law, Technology Transfer and R&D. He has Masters degrees in Business and Biomedical Engineering from UNC and Johns Hopkins, respectively.
28 April 2021
Interview

Report

Viewpoint

Accelerating the development of novel gene therapies for central nervous system diseases

Suyash Prasad, MBBS, MSc, MRCP, MRCPCH, FFPM
Suyash Prasad, MBBS, MSc, MRCP, MRCPCH, FFPM
Chief Medical Officer and Head of Research and Development, Taysha Gene Therapies
Suyash Prasad is a Pediatrician, Clinical Development Physician, Translational Scientist and Executive Leader with 20 years’ experience in the biopharmaceutical industry. He currently serves as the Chief Medical Officer and Head of Research and Development at Taysha Gene Therapies, an organization dedicated to developing AAV gene therapy approaches for treating children and adults with severe neurological disease. Suyash graduated in medicine at the University of Newcastle upon Tyne, UK where he received commendations for Pediatrics, Obstetrics and Gynecology, and Medical Ethics. His pediatric training was completed at recognized centers of excellence in the UK and Australia before he moved to industry. His industry career progressed at Eli Lilly, Genzyme, and BioMarin, and more recently he was Chief Medical Officer at the gene therapy company, Audentes Therapeutics (now Astellas Gene Therapies), before moving to Taysha. He is a UK board-certified physician and is a member of the Royal College of Physicians (MRCP) and the Royal College of Paediatrics and Child Health (MRCPCH). Suyash received his Diploma in Pharmaceutical Medicine from the Royal College of Physicians of the UK, and his Masters in Translation Science with distinction from Kings College, London. He is a Fellow of the Faculty of Pharmaceutical Medicine and is a past recipient of the Outstanding Contribution award from the Faculty. Suyash has dedicated his career to the well-being and advocacy of children who are afflicted by rare and severe disease.
19 May 2021
Viewpoint

Latest articles

Expert Roundtable Video

Embracing transformation: how big data, AI and digitization are changing cell and gene therapy manufacture

Umay Saplakoglu,
Umay Saplakoglu
Chief Digital Officer at Cytiva
Over the last 25+ years (19 of those in Cytiva) Umay had various roles in the Life Sciences mainly focusing on bioprocessing. She was the GM for Fast Trak (a global group that does process development, training and clinical manufacturing Phase 1/2 ) before taking on the lead for digital solutions team. Umay has a PhD in Biology from Middle East technical University. In her current role, Umay is leading a portfolio of digital solutions in data management, data access and in-silico modeling to help reduce waste from biopharma customers processes & accelerate CMC activities, to create value from digital.
Theresa Kotanchek,
Theresa Kotanchek
CEO at Evolved Analytics LLC
Damian Marshall,
Damian Marshall
Director, New & Enabling Technologies at Cell and Gene Therapy Catapult
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.
Krishnendu Roy,
Krishnendu Roy
Professor, Director, Marcus Center for Therapeutic Cell Characterization & Manufacturing (MC3M) at Georgia Institute of Technology
Dr. Roy’s research interests are in the areas of scalable cell manufacturing, immunoengineering, stem-cell engineering, and controlled drug and vaccine delivery technologies, with a particular focus in biomaterials. In recognition of his seminal contributions to these fields, Dr. Roy has been elected Fellow of the American Institute for Medical and Biological Engineering (AIMBE) and Fellow of the Biomedical Engineering Society (BMES). In addition, Dr. Roy has received numerous awards and honors, including Young Investigator Awards from both the Controlled Release Society (CRS) and the Society for Biomaterials (SFB), the NSF CAREER award, and the Global Indus Technovator Award from MIT, among others.He serves on the editorial boards of the Journal of Controlled Release, the European Journal of Pharmaceutics and Biopharmaceutics, the Journal of Immunology and Regenerative Medicine, and AIChE Journal of Advanced Manufacturing and Processing. He is also a member of the Forum on Regenerative Medicine of the National Academies of Science, Engineering and Medicine (NASEM) and a board member of the newly established Standards Coordinating Body (SCB) for Cell and Regenerative Therapies.
Scott Sobecki
Scott Sobecki
Director of Research Informatics at Vanderbilt- Ingram Cancer Center
14 May 2021
Expert Roundtable Video

Expert Roundtable

Embracing transformation: how big data, AI and digitization are changing cell and gene therapy manufacture

Umay Saplakoglu,
Umay Saplakoglu
Chief Digital Officer, Cytiva
Over the last 25+ years (19 of those in Cytiva) Umay had various roles in the Life Sciences mainly focusing on bioprocessing. She was the GM for Fast Trak (a global group that does process development, training and clinical manufacturing Phase 1/2 ) before taking on the lead for digital solutions team. Umay has a PhD in Biology from Middle East technical University. In her current role, Umay is leading a portfolio of digital solutions in data management, data access and in-silico modeling to help reduce waste from biopharma customers processes & accelerate CMC activities, to create value from digital.
Theresa Kotanchek,
Theresa Kotanchek
Chief Executive Officer, Evolved Analytics LLC
Damian Marshall,
Damian Marshall
Director of New Technologies, Cell and Gene Therapy Catapult
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.
Krishnendu Roy,
Krishnendu Roy
Director, Center for Immuno- Engineering, Georgia Tech
Dr. Roy’s research interests are in the areas of scalable cell manufacturing, immunoengineering, stem-cell engineering, and controlled drug and vaccine delivery technologies, with a particular focus in biomaterials. In recognition of his seminal contributions to these fields, Dr. Roy has been elected Fellow of the American Institute for Medical and Biological Engineering (AIMBE) and Fellow of the Biomedical Engineering Society (BMES). In addition, Dr. Roy has received numerous awards and honors, including Young Investigator Awards from both the Controlled Release Society (CRS) and the Society for Biomaterials (SFB), the NSF CAREER award, and the Global Indus Technovator Award from MIT, among others.He serves on the editorial boards of the Journal of Controlled Release, the European Journal of Pharmaceutics and Biopharmaceutics, the Journal of Immunology and Regenerative Medicine, and AIChE Journal of Advanced Manufacturing and Processing. He is also a member of the Forum on Regenerative Medicine of the National Academies of Science, Engineering and Medicine (NASEM) and a board member of the newly established Standards Coordinating Body (SCB) for Cell and Regenerative Therapies.
Scott Sobecki
Scott Sobecki
Director of Research Informatics, Vanderbilt- Ingram Cancer Center
14 May 2021
Expert Roundtable

Webinars

Ultrahigh-content imaging helps to identify CAR target candidates against pancreatic adenocarcinoma

Ultrahigh-content imaging helps to identify CAR target candidates against pancreatic adenocarcinoma

Dr. Daniel Schäfer
Dr. Daniel Schäfer, Team Coordinator R&D, Miltenyi Biotec
20 April 2021
Webinar
Key lessons for cell & gene therapy and mRNA therapeutic development from COVID-19 mRNA vaccine approvals

Key lessons for cell & gene therapy and mRNA therapeutic development from COVID-19 mRNA vaccine approvals

Scott Zobbi
Scott Zobbi, Senior Business Development Manager, Custom POROS Chromatography Resins, ThermoFisher Scientific
Venkata Indurthi, PhD
Venkata Indurthi, PhD, Vice President, Research and Development, Aldevron
Christoph Kröner
Christoph Kröner, Director DNA Process Development & Cap Technology, BioNTech SE
Joseph Barberio
Joseph Barberio, Director, Process Development at Strand Therapeutics Inc.
22 April 2021
Webinar