Viral vector integrity analysis 1

Viral vector integrity analysis – Advancements in gene therapy development

Gene therapy holds promise for treating various genetic diseases. In recent years, more than 30 gene therapies have been approved by the FDA, and more than 2000 are currently in development at various pipeline stages around the globe.

Recombinant Adeno-Associated Viral (AAV) vectors are widely used as a gene delivery mechanism to introduce therapeutic genes in patients. Accurate and reproducible characterization of such vectors is critical to ensure correct clinical dosing, safety, and treatment efficacy.

Genome integrity can influence vector quantitation results depending on the regions targeted for analysis. In addition, truncated vectors may not result in a functional product.

In this roundtable, our experts will discuss the available methods to assess vector integrity and the advantages dPCR-based solutions can bring. They will share perspectives on multiplex assay design to accomplish this task and related challenges, as well as discuss the importance of reference standard materials and assay qualification.

A live Q&A session will follow the discussion, during which you can ask questions to our expert panelists.

About the speaker

David Dobnik, Co-founder and Chief Scientific Officer

Niba Labs

Since 2016, David Dobnik has been closely working with companies in the field of gene therapy (such as AveXis [now Novartis Gene Therapies]) to develop and apply methods for precise quantification of viral vectors (for example, dPCR), observation of viral particles (electron microscopy), and for identification and quantification of impurities (for example nucleic acids by high-throughput sequencing [HTS/NGS] or PCR-based methods). David has been leading the development and tech-transfer projects for pharmaceutical companies focused on gene therapy, helping their process development efforts with characterizations of viral vectors. Lately, most of his work has focused on new genome integrity evaluation approaches.

Andrzej Noyszewski, PhD, MSc. Eng, Senior R&D Scientist

Agathos Biologics

Andrzej Noyszewski is a biotechnology industry professional with subject matter expertise in synthetic gene constructs, analytical methods (DNA, mRNA, proteins), downstream processing and product development. Before his role at Agathos Biologics, Noyszewski worked as a part of the Analytical Methods Validation team at Aldevron, ND. He holds a Doctor of Philosophy (PhD) in Genomics and Bioinformatics from North Dakota State University and a Master of Science and Engineering (MSc. Eng) in Biotechnology from Warsaw University of Life Sciences in Warsaw.

Alex Deiulio, B.S. Molecular Biology, Scientist

Agathos Biologics

Alex Deiulio has been with Agathos Biologics since 2022 and is a Scientist with 6 years of experience in analytical method development, validation, and implementation. His responsibilities include developing various analytical methods to characterize AAV and support cell line development. Alex leads the analytical testing team at Agathos. Alex has been focused on using the QIAcuity dPCR to characterize the genomic integrity of Agathos’ Universal AAV Standard. Alex holds a B.S. in Molecular Biology from Indian River State College.

Robert L. Hills, PhD, Director of Molecular Cell and Gene Therapies

SK pharmteco

Robert joined SK pharmteco in 2023 after serving in various roles in academia and industry. Robert is a classically trained biochemist and has contributed to developing new molecular technologies that have led to the generation of first-in-class small and large molecule entities. Before joining SK pharmteco, Robert held positions at Biomeme, The University of Pennsylvania, The Wistar Institute, Janssen, Pfizer, and the Genetics Institute.

David Eckert, Business Development Manager dPCR

QIAGEN

David earned a PhD in Biochemistry and Molecular Biology at the Louisiana State University Health Sciences Center in New Orleans. Then, he worked as a post-doc at the University of Pittsburgh. He joined Life Technologies as a Technical Applications Scientist, followed by a stint as a Field Applications Scientist, first at Bio-Rad Laboratories and then at Thermo Fisher Scientific. David is currently a Business Development Manager for digital PCR at QIAGEN.