Cell and Gene Therapies
Viral and Non-Viral Vector Gene Therapy Process Development and Optimization
From AAV to LNPs, the modern gene therapy toolbox is bursting with potential–and complexity. As these cutting-edge modalities continue to redefine what’s possible in medicine, the challenge of developing robust, scalable, and phase-appropriate manufacturing processes has never been more urgent, or more intellectually thrilling.
This session will delve into the evolving landscape of viral and non-viral vector process development, where the traditional biologics playbook and new tactics to overcome unique challenges are coalescing into modern best practice. Whether you’re grappling with lentiviral vector purification, fine-tuning an exosome-based delivery platform, or decoding the variability of full versus empty capsids, this is the forum to share your breakthroughs and battle scars.
*We invite abstracts that address:
*Innovative strategies in vector production and purification
*Process intensification and continuous manufacturing approaches
*Methods for managing complexity and heterogeneity (capsid ratios, potency, payload integrity) and insights into the impact of attributes
*Advanced analytical tools for characterizing safety, identity, and quality
*Integration of automation, big data analytics, AI/ML, and mechanistic modeling into development workflows
*Phase-appropriate CMC strategies that balance scientific rigor with regulatory pragmatism
We especially encourage case studies and real-world examples that highlight novel technologies, unexpected insights, or practical feedback. We also welcome reports of things that simply did not workâbecause sometimes failure is the most efficient process optimization of all.
Developing the future of medicine requires a bit of engineering, a dash of art, and just the right amount of controlled chaos, and this session invites all of the above. Whether you’re from academia or industry, viral or non-viral, early phase or late stage, come share how you’re helping to tame the complexity and unlock the potential of gene therapy.
Cell therapy Process Development and Optimization
Cell therapies have demonstrated their efficacy to treat or be curative in oncology and non-oncology applications. As the field continues to evolve, multiple avenues have been explored in the cellular therapy space to improve the quality and to simplify the process of generating cell therapy products, for example CAR-T, TCR-based therapies, and other gene-edited cells such as engineered NKs. This session will encompass various aspects of cell therapy process development including new rapid manufacturing processes in the autologous and iPSC approaches in the allogeneic cell therapy manufacturing space. This would include advances in the isolation, expansion, gene modification, media optimization, process scale up, analytics and characterization of cell-based therapy products. We invite topics that examine the impact of raw material, including the heterogeneity of the cell substrate, on drug product potency and purity, along with identification of predictive biomarkers and control strategies to assess and manage the impacts. Additionally, novel drug products, cryopreservation and delivery strategies for in vivo use along with improved or novel non-viral and viral vector production systems will be included in this session. Abstracts relevant to these topics are highly encouraged, including those focusing on novel process improvements, novel product designs, media development, cost minimization, control and optimization strategies, equipment and reagents design and characterization.
CMC and Regulatory Challenges
Cell and gene therapies (CGTs) have ushered in a new era of precision medicine, with several gene-modified cellular therapies, such as CAR-T cells and gene-edited hematopoietic stem cells, now achieving regulatory approvals and demonstrating remarkable clinical outcomes. Yet, CGTs continue to face substantial hurdles related to Chemistry, Manufacturing, and Controls (CMC) and evolving global regulatory landscapes. As these transformative modalities mature, challenges related to analytical characterization, product specification, process development, and raw material control are compounded by phase-appropriate development needs, safety considerations, and the complexity of global regulatory alignment. This session invites presentations that highlight cutting-edge strategies and real-world lessons in overcoming CMC and regulatory hurdles across the CGT pipeline. Topics of interest include product specification and release testing, analytical comparability and product/process lifecycle evolution, critical quality attributes (e.g., full/empty capsid content, residuals). Additionally, we are interested in process development, continuous manufacturing, control strategies (including PAT and in-process controls), and scale-up and validation approaches. We are also interested in enabling technologies that support smarter and faster development, including the use of big data, omics, machine learning, and mechanistic modeling. Contributions from both industry and academia are encouraged to share insights that can help accelerate safe, efficient, and scalable development of these advanced therapies.
Platform Approaches and Technologies
Cell and gene therapies hold immense promise in reshaping the treatment landscape, offering the potential for transformative, curative interventions. However, their manufacturing processes are still maturing and require the depth of understanding and efficiency typically associated with established biologic therapeutics. We are now witnessing the critical emergence of platform processes within the CGT space, similar to the evolution of recombinant protein therapeutics. The platform processes provide significant improvement to scalability, optimized process economics, and time-to-clinic and time-to-market. The abstracts that are invited to this session focus on cutting-edge development and production of cell therapies, gene therapies, and viral vectors, with a distinct emphasis on platform technologies. We seek abstracts that highlight innovative approaches and advancements across all the phases of development to address current manufacturing challenges within the CGT space.