The Quick Take: American Society of Cell and Gene Therapy Annual Meeting

ElevateBio’s Jimmy Xin had the opportunity to attend the American Society of Gene & Cell Therapy Annual Meeting last month and is pleased to share his perspectives about being back in person to discuss the key issues, challenges, and exciting future of cell and gene therapy research and product development.

Here’s the quick take in case you missed the meeting:

It’s been just under two weeks since the American Society of?Gene and Cell Therapy (ASGCT) Annual Meeting concluded. I am still reflecting on the incredible opportunity to join together as a community in person to discuss the key issues, challenges, and exciting future of cell and gene therapy research and product development.?

This conference enabled us to focus on the leading topics in the ever-changing landscape of cell and gene therapy. Not surprisingly, with many cell and gene therapy research programs nearing, entering, or currently in the clinic and embarking on regulatory filings, much of the talk at the conference focused on the safety and regulatory environment of these products.?

Many researchers are emphasizing process and plasmid design focused on creating a more robust and consistent product (i.e., full AAV particles). In addition, there is a focus on building out fully developed analytical panels for understanding product and impurity profiles within the process and the final product.

ElevateBio had the opportunity to present at the ASGCT conference and share the incredible work our highly-experienced lentiviral vector production team has been doing. Our team has developed a technology platform that offers our strategic partners access to suspension-based lentiviral vector production at volumes and purities that meet their needs at any stage of development, from research to cGMP manufacturing. The turnout for our poster presentation was great, suggesting the continuing need for new offerings for scientific innovators. Many people were surprised about the robustness of the upstream work and how scalable the production process is. Many scientists and other interested groups came by to ask questions about the process, development work, resources, and analytical test methods on which our team has been working.?

Capacity is limited, and demand for these services continues to grow. We believe that we can help strategic partners accelerate their development timelines, better manage costs and, above all, transform their product development and potential for clinical success so that life-changing medicines reach the patients that need them. Our lentiviral vector platform’s technological features are complemented by the holistic, end-to-end capabilities our team provides for LVV-based products -- we can help partners develop a therapeutic product from idea and IND through clinical development, regulatory submission, and cGMP manufacturing.

The keynote speakers at the conference brought up excellent perspectives about how the industry at large is shifting more toward cell and gene therapy work. Yet still, so many questions and challenges remain to be addressed – however, despite the challenges, there is a concerted and focused effort to address them together.?

Among the topics addressed by former National Institutes of Health (NIH) Director Francis Collins, M.D., Ph.D. was how addressing our ability to scale gene therapy for the future is one of the biggest obstacles facing this field to date. The ability to treat any disease anywhere in the body (including the blood/brain barrier) would lead to great strides in our ability to create meaningful therapies, but this can only be completed with in-vivo gene therapy treatments.

A discussion led by Dr. Donald Kohn from the University of California Los Angeles (UCLA) highlighted how the evolution of hematopoietic stem cell treatment for severe combined immunodeficiency (SCID) is moving toward lentiviral vector treatments, which have shown much success in patients with SCID. These advances are exactly what ElevateBio BaseCamp’s vector process development (VPD) group has been working on: creating a robust process for potent lentiviral vectors to transduce hematopoietic stem cell treatments. Dr. Kohn highlighted that future work for this type of in-vivo therapy is a platform that would allow for flexibility of this therapeutics approach with the ability to create a potential treatment for all genetic disorders – even the rarest of diseases that are often difficult to put research dollars behind.

Interested in learning more about the discussion at ASGCT or how ElevateBio has built a roadmap for future success? Hit us up at [email protected]