Bridging Science and Standards

Mr. Young: Keith, thank you for meeting with me today to discuss the development of the Lentivirus Vector Reference Material! To start out, can you tell me a bit about yourself, your background, and how you came to be a leading voice in the consortium that developed the new Lentiviral Vector Reference Material?  

Mr. Carson: I’m a chemical engineer with an MBA, and I’ve spent more than 30 years supporting the bioprocessing industry, primarily as a process development engineer. I started the Viral Vectors and Vaccines conference in 1994 and in 2000 I was invited to help coordinate the development of an Adenovirus 5 (Ad5) reference material alongside the Center for Biologics Evaluation and Research (CBER) and industry leaders. Later, in 2009, I worked on coordinating the production of the Adeno-associated virus 2 and 8 (AAV2, AAV8) reference materials, and by 2015, I was asked to coordinate the production of a lentiviral vector reference material. In all these projects, I served as the liaison between CBER and industry participants—recruiting firms to donate materials and services, ensuring regulatory comfort with every step, and managing timelines so commitments were met.

I also launched the BioProcessing Journal (BPJ) in 2002 to further support the industry in producing viral vectors and other biologics. Other than Genetic Engineering News (GEN), BPJ remains the oldest continuing publication dedicated to process development and biologics production.

Mr. Young: What inspired the creation of the Lentiviral Vector Reference Material (LVVRM), and what unmet need were you aiming to address?  

Mr. Carson: Interest in lentivirus for research and product development was growing rapidly, and both CBER and industry wanted a reference material produced using the same approach we followed for Ad5. They needed someone to act as a trusted liaison between industry and regulatory agencies, and I was asked to take on that role.

Mr. Young: How did you decide the specific design features—such as the HIV-1 backbone and VSV-G pseudotyping—for the LVVRM?  

Mr. Carson: We chose the HIV-1 backbone and VSV-G pseudotype because they are standard in all lentiviral vectors in develoment for human use. GFP was selected as the marker since it’s the most widely used for lentivirus. The VSV-G pseudotype is the dominant choice in the field, and because we opted for a stable producer cell line, the HIV-1 backbone was essentially a given. Ultimately, we based the design on a third-generation vector construct for its inherent safety. A forthcoming manuscript in BioProcessing Journal will provide a detailed discussion of the LVVRM production process for readers seeking deeper technical insight.

Mr. Young: Can you walk us through a pivotal moment during the development process that significantly shaped the final product?  

Mr. Carson: One turning point was when NRC-Canada offered to produce and purify the LVVRM, and ATCC stepped in to sterile filter, vial, store, and distribute the material. Another was working with NRC leadership to revive the project after it stalled during the pandemic.

Mr. Young: How do you envision the LVVRM transforming assay standardization and interlaboratory reproducibility in the field of gene therapy?  

Mr. Carson: For now, anyone submitting data to CBER for a lentiviral product will need to use the LVVRM. Over time, most researchers publishing lentivirus-related work will also rely on it, making it a cornerstone for standardization and reproducibility.

Mr. Young: You were also involved in the development of the Ad5, AAV2, and AAV8 reference materials. Where do you see the gene therapy industry going next? Are there any other reference materials on the horizon?  

Mr. Carson: I’m not aware of plans for additional reference materials. However, since AAV products dominate viral vector-based therapies, there may eventually be a need for reference materials for more AAV serotypes, and demand for the Ad5 reference material will certainly continue.

Mr. Young: What are your hopes for the future of gene therapy?  

Mr. Carson: My hope is that gene therapy continues to evolve into a truly transformative platform for medicine. We’re already seeing incredible progress, but the future will bring even greater precision and safety. One key area is the development of robust reference materials—particularly for Adenovirus and AAV—because these standards will accelerate innovation and regulatory confidence. I expect to see a broader range of AAV serotypes represented, enabling therapies for diverse conditions. Beyond that, gene therapy will expand its reach in cancer treatment and other complex diseases, offering solutions that were unimaginable just a decade ago.

Mr. Young: I’ve very much appreciated our discussion and your contribution to the field of gene therapy.  It’s an exciting time for gene therapy research!  Before we sign off, what advice do you have for scientists working in the gene therapy field? 

Mr. Carson: Collaboration is everything. Share knowledge, adopt reference materials, and engage with the broader community—whether through conferences, academic partnerships, or industry forums. Regulatory agencies are becoming more familiar with viral reference materials, and that’s helping the field move forward smoothly. Lentiviral vector reference materials, for example, have been needed for a long time, and their adoption will be critical for consistency and safety. Coordinated efforts across the industry will not only accelerate progress but also ensure that gene therapy fulfills its promise to patients worldwide.

Did you know?

ATCC’s viral reference materials—Adenovirus Type 5, Adeno-associated virus 2 and 8, and Lentivirus—are highly characterized for purity, particle concentration, and infectious titer, making them essential tools for standardizing gene therapy assays and ensuring reproducible results across labs.