2022 White Paper on Recent Issues in Bioanalysis: FDA Draft Guidance on Immunogenicity Information in Prescription Drug Labeling, LNP & Viral Vectors Therapeutics/Vaccines Immunogenicity, Prolongation Effect, ADA Affinity, Risk-based Approaches, NGS, qPCR, ddPCR Assays (Part 3 - Recommendations on Gene Therapy, Cell Therapy, Vaccines Immunogenicity & Technologies; Immunogenicity & Risk Assessment of Biotherapeutics and Novel Modalities; NAb Assays Integrated Approach)

Luying Pan¹, Johanna Mora², Karl Walravens³, Leslie Wagner⁴, Shirley Hopper⁵, Lina Loo⁶, David Bettoun⁷, Sarah Bond⁸, Francis Dessy³, Sean Downing¹, Fabio Garofolo⁹, Soumi Gupta¹⁰, Neil Henderson¹¹, Chad Irwin¹², Akiko Ishii-Watabe¹³, Sumit Kar¹⁴, Vibha Jawa², Julie Joseph¹², Ludovic Malvaux³, Jean-Claude Marshall¹⁵, Jessica McDevitt², Susovan Mohapatra¹⁶, Jessica Seitzer¹⁷, Justin Smith¹⁸, Therese Solstad¹⁹, Hiroshi Sugimoto¹, Omar Tounekti¹², Bonnie Wu²⁰, Yuling Wu²¹, Yuanxin Xu¹⁷, Joshua Xu²², Takenori Yamamoto¹³, Lin Yang²³, Albert Torri²⁴, Susan Kirshner⁴, Kimberly Maxfield⁴, Joao Pedras Vasconcelos⁴, Mohsen Rajabi Abhari⁴, Daniela Verthelyi⁴, Eric Brodsky⁴, Montserrat Carrasco-Triguero²⁵, John Kamerud¹⁸, Matthew Andisik²⁴, Daniel Baltrukonis¹⁸, Nicoletta Bivi²⁶, Isabelle Cludts⁵, Kelly Coble²⁷, Boris Gorovits²⁸, George R Gunn²⁹, Swati Gupta³⁰, Anders Holm Millner³¹, Alison Joyce¹⁸, Robert J Kubiak²¹, Seema Kumar³², Karen Liao³³, Mohanraj Manangeeswaran⁴, Michael Partridge²⁴, Samuel Pine³⁴, Johann Poetzl³⁵, Manoj Rajadhyaksha³⁶, Michele Rasamoelisolo³⁷, Susan Richards³⁸, Yuan Song³⁹, Steven Swanson³⁹, Seth Thacker⁴, Meenu Wadhwa⁵, Andreas Wolf⁴⁰, Lucia Zhang¹², Lin Zhou⁴

Affiliations

¹ Takeda, Cambridge, MA, USA.
² Bristol Myers Squibb, Lawrenceville, NJ, USA.
³ GlaxoSmithKline, Rixensart, Belgium.
⁴ US FDA, Silver Spring, MD, USA.
⁵ UK MHRA, London, UK.
⁶ Vertex, Boston, MA, USA.
⁷ Larimar, Bala-Cynwyd, PA, USA.
⁸ Alnylam, Cambridge, MA, USA.
⁹ BRI Frontage, Vancouver, BC, Canada.
¹⁰ BioMarin, Novato, CA, USA.
¹¹ AstraZeneca, Gothenburg, Sweden.
¹² Health Canada, Ottawa, ON, Canada.
¹³ Japan MHLW, Tokyo, Japan.
¹⁴ WRIB, Montreal, QC, Canada.
¹⁵ Moderna, Mystic, CT, USA.
¹⁶ Stoke, Cambridge, MA, USA.
¹⁷ Intellia, Cambridge, MA, USA.
¹⁸ Pfizer, Groton, CT, USA.
¹⁹ Norway NoMA, Oslo, Norway.
²⁰ Janssen R&D, Spring House, PA, USA.
²¹ AstraZeneca, Gaithersburg, MD, USA.
²² US FDA, Jefferson, AR, USA.
²³ REGENXBIO, Rockville, MD, USA.
²⁴ Regeneron, Tarrytown, NY, USA.
²⁵ Sangamo, Brisbane, CA, USA.
²⁶ Lilly, Indianapolis, IN, USA.
²⁷ Boehringer Ingelheim, Danbury, CT, USA.
²⁸ Sana Bio, Cambridge, MA, USA.
²⁹ GSK, Collegeville, PA, USA.
³⁰ AbbVie, Irvine, CA, USA.
³¹ Novo Nordisk, Maaloev, Denmark.
³² EMD Serono, Boston, MA, USA.
³³ Merck, West Point, PA, USA.
³⁴ Sanofi, Ghent, Belgium.
³⁵ Sandoz, Oberhaching, Germany.
³⁶ Alexion, New Haven, CT, USA.
³⁷ Teva, West Chester, PA, USA.
³⁸ Sanofi, Framingham, MA, USA.
³⁹ Genentech, South San Francisco, CA, USA.
⁴⁰ Amgen, Munich, Germany.

PMID: 37526071
DOI: 10.4155/bio-2023-0135

2022 White Paper on Recent Issues in Bioanalysis: FDA Draft Guidance on Immunogenicity Information in Prescription Drug Labeling, LNP & Viral Vectors Therapeutics/Vaccines Immunogenicity, Prolongation Effect, ADA Affinity, Risk-based Approaches, NGS, qPCR, ddPCR Assays (Part 3 - Recommendations on Gene Therapy, Cell Therapy, Vaccines Immunogenicity & Technologies; Immunogenicity & Risk Assessment of Biotherapeutics and Novel Modalities; NAb Assays Integrated Approach)

Luying Pan et al. Bioanalysis. 2023 Jul.

. 2023 Jul;15(14):773-814.

doi: 10.4155/bio-2023-0135. Epub 2023 Aug 1.

Authors

Affiliations

¹ Takeda, Cambridge, MA, USA.
² Bristol Myers Squibb, Lawrenceville, NJ, USA.
³ GlaxoSmithKline, Rixensart, Belgium.
⁴ US FDA, Silver Spring, MD, USA.
⁵ UK MHRA, London, UK.
⁶ Vertex, Boston, MA, USA.
⁷ Larimar, Bala-Cynwyd, PA, USA.
⁸ Alnylam, Cambridge, MA, USA.
⁹ BRI Frontage, Vancouver, BC, Canada.
¹⁰ BioMarin, Novato, CA, USA.
¹¹ AstraZeneca, Gothenburg, Sweden.
¹² Health Canada, Ottawa, ON, Canada.
¹³ Japan MHLW, Tokyo, Japan.
¹⁴ WRIB, Montreal, QC, Canada.
¹⁵ Moderna, Mystic, CT, USA.
¹⁶ Stoke, Cambridge, MA, USA.
¹⁷ Intellia, Cambridge, MA, USA.
¹⁸ Pfizer, Groton, CT, USA.
¹⁹ Norway NoMA, Oslo, Norway.
²⁰ Janssen R&D, Spring House, PA, USA.
²¹ AstraZeneca, Gaithersburg, MD, USA.
²² US FDA, Jefferson, AR, USA.
²³ REGENXBIO, Rockville, MD, USA.
²⁴ Regeneron, Tarrytown, NY, USA.
²⁵ Sangamo, Brisbane, CA, USA.
²⁶ Lilly, Indianapolis, IN, USA.
²⁷ Boehringer Ingelheim, Danbury, CT, USA.
²⁸ Sana Bio, Cambridge, MA, USA.
²⁹ GSK, Collegeville, PA, USA.
³⁰ AbbVie, Irvine, CA, USA.
³¹ Novo Nordisk, Maaloev, Denmark.
³² EMD Serono, Boston, MA, USA.
³³ Merck, West Point, PA, USA.
³⁴ Sanofi, Ghent, Belgium.
³⁵ Sandoz, Oberhaching, Germany.
³⁶ Alexion, New Haven, CT, USA.
³⁷ Teva, West Chester, PA, USA.
³⁸ Sanofi, Framingham, MA, USA.
³⁹ Genentech, South San Francisco, CA, USA.
⁴⁰ Amgen, Munich, Germany.

PMID: 37526071
DOI: 10.4155/bio-2023-0135

Abstract

The 2022 16th Workshop on Recent Issues in Bioanalysis (WRIB) took place in Atlanta, GA, USA on September 26-30, 2022. Over 1000 professionals representing pharma/biotech companies, CROs, and multiple regulatory agencies convened to actively discuss the most current topics of interest in bioanalysis. The 16th WRIB included 3 Main Workshops and 7 Specialized Workshops that together spanned 1 week in order to allow exhaustive and thorough coverage of all major issues in bioanalysis, biomarkers, immunogenicity, gene therapy, cell therapy and vaccines. Moreover, in-depth workshops on ICH M10 BMV final guideline (focused on this guideline training, interpretation, adoption and transition); mass spectrometry innovation (focused on novel technologies, novel modalities, and novel challenges); and flow cytometry bioanalysis (rising of the 3rd most common/important technology in bioanalytical labs) were the special features of the 16th edition. As in previous years, WRIB continued to gather a wide diversity of international, industry opinion leaders and regulatory authority experts working on both small and large molecules as well as gene, cell therapies and vaccines to facilitate sharing and discussions focused on improving quality, increasing regulatory compliance, and achieving scientific excellence on bioanalytical issues. This 2022 White Paper encompasses recommendations emerging from the extensive discussions held during the workshop and is aimed to provide the bioanalytical community with key information and practical solutions on topics and issues addressed, in an effort to enable advances in scientific excellence, improved quality and better regulatory compliance. Due to its length, the 2022 edition of this comprehensive White Paper has been divided into three parts for editorial reasons. This publication (Part 3) covers the recommendations on Gene Therapy, Cell therapy, Vaccines and Biotherapeutics Immunogenicity. Part 1 (Mass Spectrometry and ICH M10) and Part 2 (LBA, Biomarkers/CDx and Cytometry) are published in volume 15 of Bioanalysis, issues 16 and 15 (2023), respectively.

Keywords: WRIB; bioanalysis; biomarkers; cell therapy; gene therapy; immunogenicity; vaccine.

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