2019 White Paper on Recent Issues in Bioanalysis: FDA Immunogenicity Guidance, Gene Therapy, Critical Reagents, Biomarkers and Flow Cytometry Validation (Part 3 - Recommendations on 2019 FDA Immunogenicity Guidance, Gene Therapy Bioanalytical Challenges, Strategies for Critical Reagent Management, Biomarker Assay Validation, Flow Cytometry Validation & CLSI H62)

Steven Piccoli¹, Devangi Mehta², Alessandra Vitaliti³, John Allinson⁴, Shashi Amur⁵, Steve Eck⁶, Cherie Green⁷, Michael Hedrick⁸, Shirley Hopper⁹, Allena Ji¹⁰, Alison Joyce¹¹, Virginia Litwin¹², Kevin Maher⁵, Joel Mathews¹³, Kun Peng⁷, Afshin Safavi¹⁴, Yow-Ming Wang⁵, Yan Zhang⁸, Lakshmi Amaravadi¹⁵, Nisha Palackal¹⁶, Sai Thankamony⁸, Chris Beaver¹⁷, Eris Bame², Thomas Emrich¹⁸, Christine Grimaldi¹⁹, Jonathan Haulenbeek⁸, Alison Joyce¹¹, Vellalore Kakkanaiah²⁰, David Lanham²¹, Kevin Maher⁵, Andrew Mayer¹, Paul C Trampont²², Laurent Vermet²³, Naveen Dakappagari²⁴, Catherine Fleener²⁵, Fabio Garofolo²⁶, Cynthia Rogers²⁷, Shabnam Tangri²⁴, Yuanxin Xu²⁸, Meina Liang²⁹, Manoj Rajadhyaksha¹⁶, Susan Richards¹⁰, Becky Schweighardt³⁰, Shobha Purushothama², Daniel Baltrukonis³¹, Jochen Brumm⁷, Elana Cherry³², Jason Delcarpini³³, Carol Gleason⁸, Susan Kirshner⁵, Robert Kubiak⁶, Luying Pan³⁴, Michael Partridge¹⁶, João Pedras-Vasconcelos⁵, Qiang Qu³⁵, Venke Skibeli³⁶, Therese Solstad Saunders³⁶, Roland F Staack¹⁸, Kay Stubenrauch¹⁸, Al Torri¹⁶, Daniela Verthelyi⁵, Haoheng Yan⁵, Boris Gorovits¹¹, Rachel Palmer¹⁰, Mark Milton³⁷, Brian Long³⁰, Bart Corsaro³⁸, Vahid Farrokhi¹¹, Michele Fiscella³⁹, Neil Henderson⁴⁰, Vibha Jawa⁴¹, Jim McNally³⁴, Rocio Murphy⁴², Hanspeter Waldner⁴³, Tong-Yuan Yang⁴⁴

Affiliations

¹ GlaxoSmithKline, Collegeville, PA, USA.
² Biogen, Cambridge, MA, USA.
³ Novartis Pharma, Basel, Switzerland.
⁴ Immunologix Laboratories, Tampa, FL, USA.
⁵ US FDA, Silver Spring, MD, USA.
⁶ AstraZeneca, Gaithersburg, MD, USA.
⁷ Genentech, South San Francisco, CA, USA.
⁸ Bristol-Myers Squibb, Princeton, NJ, USA.
⁹ UK MHRA-NIBSC, London, UK.
¹⁰ Sanofi, Framingham, MA, USA.
¹¹ Pfizer, Andover, MA, USA.
¹² Caprion Biosciences, Montreal, QC, Canada.
¹³ Ionis Pharmaceutical, Carlsbad, CA, USA.
¹⁴ BioAgilytix, Durham, NC, USA.
¹⁵ Shire/Takeda, Lexington, MA, USA.
¹⁶ Regeneron Pharmaceuticals, Tarrytown, NY, USA.
¹⁷ Syneos Health, Princeton, NJ, USA.
¹⁸ Roche Pharma Research & Early Development, Roche Innovation Center, Munich, Germany.
¹⁹ Boehringer Ingelheim, Ridgefield, CT, USA.
²⁰ PPD, Richmond, VA, USA.
²¹ Eurofins Bioanalysis Services, Abingdon, UK.
²² Covance Central Laboratory, Greenfield, IN, USA.
²³ Sanofi, Montpellier, France.
²⁴ Navigate, a Novartis Subsidiary, San Diego, CA, USA.
²⁵ Pfizer, San Diego, CA, USA.
²⁶ Angelini Pharma, Pomezia, RM, Italy.
²⁷ bluebird bio, Cambridge, MA, USA.
²⁸ Alnylam Pharmaceuticals, Cambridge, MA, USA.
²⁹ AstraZeneca, South San Francisco, CA, USA.
³⁰ BioMarin, Novato, CA, USA.
³¹ Pfizer, Groton, CT, USA.
³² Health Canada, Ottawa, ON, Canada.
³³ Celldex Therapeutics, Needham, MA, USA.
³⁴ Shire/Takeda, Cambridge, MA, USA.
³⁵ EMD Serono/Merck KGaA, Billerica, MA, USA.
³⁶ Norwegian Medicines Agency, Oslo, Norway.
³⁷ Novartis, Cambridge, MA, USA.
³⁸ GlaxoSmithKline, Rockville, MD, USA.
³⁹ REGENXBIO, Rockville, MD, USA.
⁴⁰ AstraZeneca, Molndal, Sweden.
⁴¹ Merck & Co., Inc., Kenilworth, NJ, USA.
⁴² Merck & Co., Inc., West Point, PA, USA.
⁴³ CRISPR Therapeutics, Cambridge, MA, USA.
⁴⁴ Janssen R&D, Spring House, PA, USA.

PMID: 31820675
DOI: 10.4155/bio-2019-0271

2019 White Paper on Recent Issues in Bioanalysis: FDA Immunogenicity Guidance, Gene Therapy, Critical Reagents, Biomarkers and Flow Cytometry Validation (Part 3 - Recommendations on 2019 FDA Immunogenicity Guidance, Gene Therapy Bioanalytical Challenges, Strategies for Critical Reagent Management, Biomarker Assay Validation, Flow Cytometry Validation & CLSI H62)

Steven Piccoli et al. Bioanalysis. 2019 Dec.

. 2019 Dec;11(24):2207-2244.

doi: 10.4155/bio-2019-0271. Epub 2019 Dec 10.

Authors

Affiliations

¹ GlaxoSmithKline, Collegeville, PA, USA.
² Biogen, Cambridge, MA, USA.
³ Novartis Pharma, Basel, Switzerland.
⁴ Immunologix Laboratories, Tampa, FL, USA.
⁵ US FDA, Silver Spring, MD, USA.
⁶ AstraZeneca, Gaithersburg, MD, USA.
⁷ Genentech, South San Francisco, CA, USA.
⁸ Bristol-Myers Squibb, Princeton, NJ, USA.
⁹ UK MHRA-NIBSC, London, UK.
¹⁰ Sanofi, Framingham, MA, USA.
¹¹ Pfizer, Andover, MA, USA.
¹² Caprion Biosciences, Montreal, QC, Canada.
¹³ Ionis Pharmaceutical, Carlsbad, CA, USA.
¹⁴ BioAgilytix, Durham, NC, USA.
¹⁵ Shire/Takeda, Lexington, MA, USA.
¹⁶ Regeneron Pharmaceuticals, Tarrytown, NY, USA.
¹⁷ Syneos Health, Princeton, NJ, USA.
¹⁸ Roche Pharma Research & Early Development, Roche Innovation Center, Munich, Germany.
¹⁹ Boehringer Ingelheim, Ridgefield, CT, USA.
²⁰ PPD, Richmond, VA, USA.
²¹ Eurofins Bioanalysis Services, Abingdon, UK.
²² Covance Central Laboratory, Greenfield, IN, USA.
²³ Sanofi, Montpellier, France.
²⁴ Navigate, a Novartis Subsidiary, San Diego, CA, USA.
²⁵ Pfizer, San Diego, CA, USA.
²⁶ Angelini Pharma, Pomezia, RM, Italy.
²⁷ bluebird bio, Cambridge, MA, USA.
²⁸ Alnylam Pharmaceuticals, Cambridge, MA, USA.
²⁹ AstraZeneca, South San Francisco, CA, USA.
³⁰ BioMarin, Novato, CA, USA.
³¹ Pfizer, Groton, CT, USA.
³² Health Canada, Ottawa, ON, Canada.
³³ Celldex Therapeutics, Needham, MA, USA.
³⁴ Shire/Takeda, Cambridge, MA, USA.
³⁵ EMD Serono/Merck KGaA, Billerica, MA, USA.
³⁶ Norwegian Medicines Agency, Oslo, Norway.
³⁷ Novartis, Cambridge, MA, USA.
³⁸ GlaxoSmithKline, Rockville, MD, USA.
³⁹ REGENXBIO, Rockville, MD, USA.
⁴⁰ AstraZeneca, Molndal, Sweden.
⁴¹ Merck & Co., Inc., Kenilworth, NJ, USA.
⁴² Merck & Co., Inc., West Point, PA, USA.
⁴³ CRISPR Therapeutics, Cambridge, MA, USA.
⁴⁴ Janssen R&D, Spring House, PA, USA.

PMID: 31820675
DOI: 10.4155/bio-2019-0271

Abstract

The 2019 13^th Workshop on Recent Issues in Bioanalysis (WRIB) took place in New Orleans, LA, USA on April 1-5, 2019 with an attendance of over 1000 representatives from pharmaceutical/biopharmaceutical companies, biotechnology companies, contract research organizations and regulatory agencies worldwide. WRIB was once again a 5-day, week-long event - a full immersion week of bioanalysis, biomarkers, immunogenicity and gene therapy. As usual, it was specifically designed to facilitate sharing, reviewing, discussing and agreeing on approaches to address the most current issues of interest including both small- and large-molecule bioanalysis involving LCMS, hybrid LBA/LCMS, LBA cell-based/flow cytometry assays and qPCR approaches. This 2019 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 2019 edition of this comprehensive White Paper has been divided into three parts for editorial reasons. This publication (Part 3) covers New Insights in Biomarker Assay Validation, Current & Effective Strategies for Critical Reagent Management, Flow Cytometry Validation in Drug Discovery & Development & CLSI H62, Interpretation of the 2019 FDA Immunogenicity Guidance and Gene Therapy Bioanalytical Challenges. Part 1 (Innovation in Small Molecules and Oligonucleotides & Mass Spectrometry Method Development Strategies for Large Molecule Bioanalysis) and Part 2 (Recommendations on the 2018 FDA BMV Guidance, 2019 ICH M10 BMV Draft Guideline and regulatory agencies' input on bioanalysis, biomarkers, immunogenicity and gene therapy) are published in volume 11 of Bioanalysis, issues 22 and 23 (2019), respectively.

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