The conference segment on Breaking Biological Barriers opened with Jennifer Johnston of NysnoBio emphasizing how rare diseases may not be so rare after all, advocating for broader targeting approaches in therapeutic development. In the Beyond Immunity section, Professor Niren Murthy of UC Berkeley presented innovative methods to reduce host immune responses while enhancing viral gene therapy delivery efficiency, showcasing advances in vector design and stability. The Advancing CRISPR Precision panel followed, featuring a series of talks focused on addressing the persistent challenge of off-target effects in CRISPR-Cas9 genome editing. Speakers including Xiaoya Sun of Takeda and Brad Ringeisen of the Innovative Genomics Institute highlighted novel Cas9 variants and computational tools aimed at improving editing specificity and safety. A robust panel discussion on CAR-T and TCR-T Therapies in Solid Tumors then explored strategies for overcoming translational hurdles, optimizing persistence, and personalizing approaches using biomarkers and combination therapies. In the Overcoming Variability session, scalable manufacturing of cell-based therapies was addressed with a focus on quality, cost-efficiency, and regulatory compliance. The afternoon concluded with a presentation by Frederic Padilla from the Focused Ultrasound Foundation, detailing the promise of focused ultrasound (FUS) as a non-invasive platform for delivering gene and cell therapies across the blood-brain barrier, with implications for broader organ targeting and clinical translation.

The afternoon sessions began with a focus on driving clinical research forward through digital innovation and strategic service integration. This session highlighted how Clinical Central Services are leveraging emerging technologies to enhance research accessibility, diversity, and operational efficiency, ultimately accelerating clinical trial execution. Following a Q&A and networking break, the Clinical Trials Challenges Panel delved into critical issues facing cell and gene therapy trials. Experts discussed enhancing eligibility precision and overcoming immune and disease-stage challenges, especially in the context of HLA matching. Subsequent talks addressed the complexity of clinical trial design in rare disease populations, emphasizing innovative approaches to endpoints and regulatory compliance. The conversation progressed to long-term follow-up considerations, focusing on balancing safety, efficacy, and cost. The final sessions explored risk-based monitoring strategies tailored to CGT protocols, with insights into real-time analytics, regulatory expectations, and patient safety. The day concluded with a discussion on implementing real-time data capture and analytics in CGT operations, showcasing how digital platforms are transforming protocol adherence, trial logistics, and operational excellence. Panelists included leaders from Eli Lilly and Emmes, sharing valuable perspectives from the forefront of CGT research.

On Day 2 of the conference, the Cell & Gene Therapy Manufacturing Panel Discussion offered a comprehensive exploration of innovative strategies aimed at addressing bottlenecks in the production of advanced therapies. The session opened with Chaozhong Zou (Cincinnati Children's) discussing critical product and process challenges, emphasizing methods to streamline workflows and enhance batch consistency. This was followed by insights into fully automated, closed-system technologies, which improve scalability, contamination control, and regulatory compliance. The panel then examined scalable viral vector manufacturing and the integration of modular facility design to enable agile, multi-product environments, highlighting the transition from clinical to commercial production. Standardization of potency assays was addressed as a cornerstone for ensuring clinical consistency across autologous and allogeneic therapies. A focused discussion on allogeneic cell therapies showcased scalable, off-the-shelf manufacturing strategies and the role of gene editing in overcoming immunogenicity challenges. The morning concluded with a talk on redefining manufacturing excellence through operational innovation, including advanced automation, QbD approaches, and data-driven process control. Panelists—Zou, Hao Liu (UMass), and Kok-Seong Lim—shared practical insights before a networking break. The afternoon resumed with sessions emphasizing scalable, safe, and sustainable manufacturing strategies, highlighting cutting-edge technologies such as real-time monitoring and eco-conscious processes that are transforming the future of cell and gene therapy manufacturing.
 

The afternoon sessions of Day 2 delved into the critical intersection of innovation, market access, and reimbursement strategies in the rapidly evolving field of cell and gene therapy (CGT). Beginning with a strategic discussion on aligning innovation with infrastructure to accelerate global CGT commercialization, participants explored actionable frameworks for broadening patient access and streamlining launch timelines. This was followed by a focused session on the reimbursement challenges faced by fee-for-service Medicaid programs, highlighting innovative strategies employed by states to sustain access and manage costs. A dynamic panel discussion examined value-based pricing models and the integration of real-world evidence (RWE) to enhance payer confidence. Subsequent presentations addressed the role of AI in bridging access gaps, the importance of early stakeholder engagement, and the promise and pitfalls of indication-based pricing. The day concluded with an in-depth look at designing outcome-based agreements that balance payer risk and biotech returns, emphasizing the need for adaptable, evidence-driven strategies to ensure market success.

The 2025 Cell & Gene Therapy Summit will be a landmark event that brings together leading experts to discuss the future of gene therapies. The focus on smarter vectors, improved CRISPR precision, and scalable manufacturing reflects the cutting-edge nature of the field, and attendees will leave with a deeper understanding of the challenges and opportunities ahead. As the science of gene therapy continues to evolve, the summit will showcase the advancements needed to move from breakthrough research to transformative clinical applications that can revolutionize the treatment of genetic and acquired disorders.