Rich R. Pelt

Senior Director, Vaccines CMC Regulatory Affairs

Andrew S. Nelson

Manager Regulatory CMC Strategy – Vaccines

Michael Parks

Director Regulatory GCMC Vaccines

Adrienne K. Stafford

Vaccines Director, CMC Regulatory

Pfizer, Inc.

Fireside Chat: Regulatory Challenges During the Pandemic

Fouad Atouf, PhD

Vice President, Global Biologics

US Pharmacopeial Convention (USP)

mRNA — Analytical Solutions to Support Adoption of This Modality

Bio

Wei-Chiang Chen, PhD

Associate Director, BioProcess Analytics, Genomic Medicine Unit

Sanofi US

Establishing a High-Throughput CE-SDS Platform for Multiple Applications on Gene Therapy Analytics

Abstract

Samantha Maragh, PhD

Leader, Genome Editing Program

National Institute of Standards & Technology (NIST)

Sophie Sichel, PhD

Scientist, Non-Viral Delivery, Gene Delivery Process and Analytical Development

Bristol-Myers Squibb Company

LNP Formulation and Non-Viral Gene Delivery for CAR T-cells

John Nelson, PhD

Senior Principal Scientist

GE Research

Rolling Circle Amplification

Peter Dedon, MD, PhD

Singapore Professor, Department of Biological Engineering; Lead Principal Investigator — Singapore-MIT Alliance for Research and Technology (SMART) Antimicrobial Resistance Interdisciplinary Research Group (AMR IRG)

Massachusetts Institute of Technology (MIT)

New Tools for Discovering and Mapping Nucleic Acid Modifications

Abstract Bio

Jeffrey Wilusz, PhD

Professor — Department of Microbiology, Immunology and Pathology

Colorado State University

RNA Wars: RNA Viruses Versus the RNA Biology of the Cell

Abstract Bio

Marcell Veidner

Business Development Manager

Genedata, Inc.

Bioinformatics

Bio

Tim Morgan

Business Development Manager, Formulation and Drug Delivery

MilliporeSigma

Khaled Yamout

Senior Director of Quality Control

TriLink BioTechnologies, part of Maravai LifeSciences

Fouad Atouf

Fouad Atouf is Vice President, Science—Global Biologics for USP. He leads all scientific activities related to the development and maintenance of documentary and reference standards for biotherapeutics and advanced therapies. Prior to joining USP in 2006, his research at the US National Institutes of Health (NIH) focused on developing methods for the development of cell-based therapies for the treatment of diabetes. Dr. Atouf earned his PhD in Cell Biology from the Pierre and Marie Curie University, Paris, France.

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Establishing a High-Throughput CE-SDS Platform for Multiple Applications on Gene Therapy Analytics

Gene therapy has been demonstrated as a promising technology in the biotechnology industry. A therapeutic sequence is delivered via either viral product or lipid nanoparticle to specific human tissues and creates long term therapeutic effects. In this presentation, we present the work on developing a high-0throughput CE-SDS platform for both viral and non-viral gene therapy applications. For adeno-associated virus (AAV) gene therapy, our platform method is capable of monitoring multiple quality attributes in one run. For non-viral gene therapy, the established platform can be applied to analyze the integrity of the nucleic acid payloads, quantify any potential impurities, as well as understand lipid content. The high-throughput CE-SDS platform is a great tool for process development, process optimization, and manufacturing control.

REV 111422
New Tools for Discovering and Mapping Nucleic Acid Modifications

Naturally occurring epigenetic and epitranscriptomic modifications of nucleic acids offer a new dimension in therapeutics and biotechnology. As intentional components of RNA and DNA therapeutics, these chemical modifications control pharmacodynamics, pharmacokinetics, and toxicity. At the same time, DNA and RNA molecules are sensitive to adventitious modification (i.e., damage) during manufacturing. Both intentional and unintentional nucleic acid modifications pose challenges for therapeutic discovery, development, and CMC. Here I present new mass spectrometry-based (MS) tools for discovering, quantifying, and mapping DNA and RNA modifications of all types. Untargeted MS-based analysis of the “adductome” in DNA and RNA reveals dozens of damage products unique to the nucleic acid environment, with the potential for adductomic patterns to serve as QC benchmarks and for simple workflows to identify specific adducts. MS-based tools for both quantification and sequence mapping of intentional DNA and RNA modifications can also be applied at all stages of therapeutic development and manufacturing. These new tools provide an opportunity to deconstruct the pig’s breakfast of damage and modifications confronted by the nucleic acid therapeutics industry.

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Peter Dedon, MD, PhD

Pete Dedon is the Singapore Professor in the Department of Biological Engineering at MIT and the Lead Principal Investigator in the Singapore-MIT Alliance for Research and Technology Antimicrobial Drug Resistance IRG. With a research program that applies chemical approaches to nucleic acid biology, his group has developed a variety of analytical and informatics platforms for basic and translational research in epigenetics, epitranscriptomics, and genetic toxicology in infectious disease and cancer. One platform coordinates new sequencing technologies, comparative genomics, and mass spectrometry to discover novel epigenetic marks, such as phosphorothioate, hypoxanthine, and 7-deazaguanine modifications in bacterial and bacteriophage genomes in the human microbiome. In the realm of the epitranscriptome — the dozens of modified ribonucleosides in all forms of RNA — his team has developed and applied systems-level analytics to discover a mechanism of translational regulation of gene expression in bacteria, parasites, mammalian cells, and humans. This response mechanism coordinates stress-specific reprogramming of 40–50 different tRNA modifications with alternative genetic information consisting of biased used of synonymous codons in families of stress response genes. The net result is selective translation of codon-based gene families essential for survival or phenotypic change. Pete and colleagues are leveraging these discoveries to develop new enzymatic tools for biotechnology, new methods for industrial microbiology and protein production, and novel antimicrobial agents and biological therapeutics.

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RNA Wars: RNA Viruses Versus the RNA Biology of the Cell

RNA viruses usurp, avoid, and generally wreak havoc on the RNA biology of the infected cell. Understanding the impact of viral RNA in the infected cell as well as the underlying mechanisms, therefore will undoubtedly inform the design of safe and maximally effective RNA therapeutics. We have investigated a variety of mechanisms that RNA viruses use to interface with the process of mRNA stability, splicing, polyadenylation, and modification in infected cells. Both specific sequences and structures of the introduced RNA play a role in this interplay with cellular RNA biology. This work has illustrated a variety of important considerations that should be taken into account when RNA is introduced into cells. Notably, for example, the impact on the RNA biology of the cell through the sequestration of cellular RNA binding proteins by abundant viral RNAs can be very significant. Ultimately, translating these mechanistic findings into RNA therapeutic design may be an important considering for this rapidly developing field.

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Jeffrey Wilusz, PhD

Jeff Wilusz is a Professor in the Department of Microbiology, Immunology, and Pathology at Colorado State University. When not rooting for the Denver Broncos, his research interests include understanding the interface between viruses and the RNA biology of the cell. He is an elected fellow of the American Association for the Advancement of Science (AAAS), Editor-in-Chief of the RNA review journal WIREs-RNA, an honorary lifetime member of the American Society for Virology, and is a past President of the Rocky Mountain Branch of the American Society of Microbiology.

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Marcell Veidner

Marcell Veidner is Business Development Manager at Genedata. He joined the company in 2017 and has been working with global biopharma companies and emerging biotechs in implementing NGS to improve productivity, quality, and safety across cell line process development and manufacturing. Prior to joining Genedata, Marcell spun-out from Northeastern University and Harvard Medical School and served as founding CEO at Scipher Medicine, a precision immunology company matching each patient with their most effective therapy. Earlier in his career, Marcell worked as a biopharma management consultant in various firms in Europe and the US. He received his master’s degree in Biotechnology Management from the joint program of IE Business School, Madrid and Harvard Business School, Boston.

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