Main Conference: September 23-25, 2026 | Boston, MA
Main Conference Track: Development of Emerging Cell & Gene Therapies
Deep dive into the breakthroughs shaping the next era of advanced therapies
Engineering the Next Wave of Cell & Gene Therapies
Blurb: The Development of Emerging Cell and Gene Therapies track is a 2-day deep dive into the next generation of cell and gene therapies expanding beyond rare diseases into solid tumours and larger patient populations. This track delivers real-world case studies on scaling in vivo CAR-T therapies, AI-driven product design, disruptive targeted delivery technologies, and leveraging gene editing tools for in vivo delivery and applications beyond the liver, enhancing precision and therapeutic potential. Hear from industry pioneers on next-generation non-viral delivery systems and scalable solutions that are redefining the future of cell and gene therapies.
Session Spotlight: In Vivo Therapies: From Concept to Reality
Keynote: Programming Immunity In Vivo: Targeted RNA–LNP Platforms for Precision CAR Engineering
Friday, September 25th 2026 09:30am
Robert Hofmeister, Chief Scientific Officer at CREATE Medicines
This keynote presentation explores how CREATE Medicines is developing in vivo CAR therapies using targeted mRNA–LNP delivery to program immune cells directly in patients. By combining modular CAR design with optimized RNA architectures, the platform enables durable, controlled expression across T cells and innate compartments. Preclinical and clinical data demonstrate robust activity of in vivo engineered CAR-T cells, supporting scalable, off-the-shelf immunotherapies for autoimmune diseases and cancer.
Track Themes: Building the Future - A CGT Development Blueprint
Navigating the Road Ahead for In Vivo CAR-T
Explore critical product development, clinical advances, scale-up, delivery, and manufacturing strategies for in vivo CAR-T therapies, addressing production challenges, regulatory considerations, and the future potential of this transformative approach.
Targeted Delivery & Gene Editing
Go beyond viral vectors and explore the manufacturing and CMC challenges for in vivo gene editing and LNP-based delivery systems enabling enhanced efficiency, precision, and therapeutic reach beyond hepatic targets.
Starting with the End in Mind
Uncover strategic approaches to designing smart scalable, phase-appropriate CGT manufacturing processes that balance early-stage flexibility with long-term commercial viability, regulatory compliance, and broad patient access.
R&D Innovations
Discover cutting-edge R&D developments including, in vivo CAR-T, solid tumours, and new therapeutic modalities expanding treatments to broader populations.
Learn from Leading R&D Visionaries Accelerating Innovation in Cell and Gene Therapy
Explore the full Development of Emerging Cell and Gene Therapies Agenda
Development of Emerging Cell & Gene Therapies: Q&A & Expert Insights
What are the unique manufacturing challenges for in vivo CAR-T therapies compared to ex vivo approaches?
In vivo CAR-T therapies present distinct manufacturing challenges centered on viral vector production and delivery systems. Unlike ex vivo CAR-T requiring patient-specific processing, in vivo approaches demand scalable, off-the-shelf vector manufacturing for systemic administration. Critical challenges include achieving sufficient vector titers for effective in-body T-cell transduction, developing targeting mechanisms to direct gene delivery specifically to T-cells while avoiding off-target tissues, and managing immunogenicity risks. Manufacturing strategies must address large-scale AAV or LV vector production, formulation stability for distribution, and robust analytics to characterize vector potency and safety profiles.
What are the key challenges in LNP manufacturing for in vivo delivery?
Manufacturing Lipid Nanoparticles (LNPs) requires precise control over Critical Process Parameters (CPPs) to ensure consistent particle size and efficient encapsulation of the payload (e.g., mRNA or gene-editing tools). Scaling these processes from the lab to GMP requires robust strategies to maintain particle attributes and stability for effective in vivo delivery.
How should manufacturing strategies differ across clinical development phases for emerging cell and gene therapies?
Phase-appropriate manufacturing requires balancing flexibility, cost, and scalability throughout development. Early-phase manufacturing should prioritize speed and flexibility using modular, small-scale systems that accommodate protocol changes without significant capital investment. Mid-phase development demands process optimization and preliminary scale-up, implementing platform technologies and beginning CDMO discussions. Late-phase and commercial manufacturing requires fully validated, scalable processes with established supply chains. Key considerations include right-sizing facility investments to avoid over-capitalization, implementing process analytical technologies early to facilitate seamless scale-up, and maintaining comparability through development stages to minimize regulatory burden.
How is artificial intelligence transforming the development and manufacturing of cell and gene therapies?
AI is revolutionizing cell and gene therapy development through predictive modeling, process optimization, and accelerated design cycles. Machine learning algorithms analyze vector engineering datasets to predict capsid variants with improved tissue tropism and reduced immunogenicity, significantly reducing experimental iterations. In manufacturing, AI-driven process analytical technology enables real-time quality prediction and adaptive process control, optimizing parameters to maximize yield and quality. AI also accelerates analytical method development by identifying critical quality attributes and their relationships to clinical outcomes. Additionally, natural language processing tools mine scientific literature and regulatory databases to inform development strategies and identify potential safety signals earlier, ultimately reducing timelines and costs.