Innovative Idea Generating Centre
CELL & GENE THERAPY SUMMIT 2025
Redefining Possibilities Through Smarter Delivery, Safer Editing, and Scalable Innovation
DATE: 12th November , 2025 2025(Eastern Time) | Virtual Congress
BREAKING BIOLOGICAL BARRIERS
09:00 Engineering Smarter Vectors To Revolutionize Precision Delivery Of Genetic Therapies With Unmatched Specificity And Clinical Impact.
- Explore the next frontier in genetic medicine: precision delivery of therapeutic payloads to specific cells and tissues.
- Discuss cutting-edge strategies in vector engineering to overcome biological delivery barriers.
- Highlight approaches that enhance specificity, efficiency, and safety in genetic therapy delivery.
- Provide insights into the rational design of next generation viral and non-viral vectors.
- Showcase tailored delivery systems for a broad range of therapeutic applications.
- Emphasize translational breakthroughs bridging the gap from bench to bedside.
- Present how smarter vector systems are enabling more effective and clinically impactful gene therapies.
09:25 Questions & Discussion
BEYOND IMMUNITY
09:30 REDUCING HOST RESPONSES WHILE ENHANCING DELIVERY IN VIRAL GENE THERAPY PLATFORMS
Viral gene therapy has emerged as a promising avenue for the treatment of a variety of genetic disorders. However, the success of these therapies often hinges on two critical challenges: the immune responses of the host and the efficiency of viral delivery systems. This talk will delve into innovative strategies designed to reduce host immune responses to viral vectors while simultaneously enhancing their ability to effectively deliver genetic material to targeted cells. Key areas of focus include:
- Exploration of genetic modifications and novel viral platform designs aimed at minimizing immune recognition and improving vector stability and specificity.
- Discussion on advancements in vector delivery techniques, such as tissue-specific targeting and controlled release, to increase the therapeutic efficacy of gene therapies.
- Examination of current clinical trials and preclinical research that showcase these advancements, along with a forward-looking perspective on overcoming remaining obstacles.
Niren Murthy Professor UC Berkeley
09:55 Questions & Discussion
ADVANCING CRISPR-CAS9 PRECISION PANEL DISCUSSION
10:00 CRISPR Under the Microscope: Innovations for Precision and Safety in Genome Editing
10:00 Navigating the Precision and Pitfalls of CRISPR-CAS9: Addressing Off-Target Effects in Genome Editing.
CRISPR-Cas9 revolutionizes gene editing with unprecedented precision, but off-target effects remain a critical challenge. This talk explores strategies to enhance specificity, ensuring safer, more effective applications in medicine and biotechnology.
Xiaoya Sun Scientist, Analytical Development, Cell Therapy Takeda
10:15 Off-Target Effects in CRISPR: A Critical Barrier to Reliable Therapeutic Applications.
This talk explores the significance of off-target effects in CRISPR technology, discussing their impact on therapeutic interventions advancements. We’ll examine current challenges, potential risks, and strategies for improving precision.
10:30 Unveiling Off-Target Modifications: Advancing CRISPR Safety in Medicine.
Understanding off-target modifications is crucial for enhancing CRISPR's safety. Investigating unintended modifications requires exploring the mechanisms driving off-target effects to improve precision and reduce risks.
10:45 Enhancing CRISPR Precision and Safety: Strategies for Detecting and Mitigating Off-Target Effects with Novel Cas9 Variants and Computational Algorithms
This session offers an in-depth look at the latest strategies aimed at improving CRISPR technology by detecting and mitigating off-target effects. We will discuss novel Cas9 variants and computational algorithms crucial for reducing unintended genetic modifications, ensuring safer gene editing.
11:00 Enhancing Precision: The Future of CRISPR Technology in Genetic Engineering
Advancements in CRISPR technology are significantly improving its accuracy, reducing off-target effects, and advancing safer genetic engineering practices. Future innovations aim to refine specificity, paving the way for broader, more reliable applications in medicine.
11:15 Panelist:
Brad Ringeisen Executive Director Innovative Genomics Institute
Xiaoya Sun Scientist, Analytical Development, Cell Therapy Takeda
11:30 Refreshment Break
CAR-T and TCR-T THERAPIES
12:00 Enhancing CAR-T and TCR-T Therapies in Solid Tumors: Clinical Insights on Personalization, Combination Approaches, and Translational Hurdles
- Advancing Clinical Strategies for CAR-T and TCR-T Cell Therapies in Solid Tumors: Overcoming Challenges and Enhancing Efficacy
- CAR-T and TCR-T Cell Therapies in Solid Tumors: Navigating Safety, Persistence, and Tumor Microenvironment Challenges in Clinical Trials
- Optimizing CAR-T and TCR-T Cell Therapies: Lessons from Clinical Trials and Future Directions for Solid Tumor Treatment
- Personalizing CAR-T and TCR-T Therapies for Solid Tumors: Biomarkers, Combination Strategies, and Clinical Translation Challenges
12:25 Questions & Discussion
OVERCOMING VARIABILITY
12:30 Scalable Manufacturing Strategies For Cell Based Therapies To Achieve Quality, Affordability, And Regulatory Excellence In Advanced Bioproduction
Scaling the production of cell-based therapies poses significant technological and economic challenges. Ensuring consistent quality across batches is difficult due to inherent product variability. This talk explores innovative strategies to enhance manufacturing scalability while maintaining rigorous quality control, addressing key hurdles in standardization, cost-efficiency, and regulatory compliance in advanced therapeutic production.
12:55 Questions & Discussion
13:00 Lunch Break
PANEL DISCUSSION
Innovative Gene Therapy Clinical Trial Designs Improving Efficiency Data Integrity and Accelerating Development Timelines
14:00 Bridging Preclinical Insights with Clinical Execution for Accelerated Success in Cell and Gene Therapy Trials
This session will explore how early-stage insights can be strategically aligned with clinical trial design, regulatory pathways, and patient-centric considerations to accelerate therapeutic success. Attendees will gain perspectives on optimizing preclinical models, anticipating translational hurdles, and implementing adaptive clinical strategies that reduce risk and enhance trial efficiency. Real-world case studies will illustrate how integrated cross-functional planning can shorten development timelines and increase the likelihood of clinical impact.
14:15 Early Detection and Mitigation of Operational and Clinical Threats to Ensure Success in Cell and Gene Therapy Trials through Proactive Risk-Based Quality Management
In this presentation, we’ll investigate how implementing proactive, risk-based quality management strategies can identify and address operational and clinical threats early in cell and gene therapy trials. By leveraging data-driven risk assessment and mitigation techniques, sponsors and clinical teams can enhance trial integrity, patient safety, and regulatory compliance. Attendees will gain insights into practical approaches for minimizing disruptions, optimizing trial outcomes, and accelerating the development of innovative therapies in this rapidly
evolving field.
14:30 Collaborative Trial Networks and CRO Partnerships Driving Innovation to Enhance Efficiency and Accelerate Clinical Trial Success
This talk explores how strategic collaboration between trial networks and Contract Research Organizations (CROs) is transforming clinical research. By leveraging innovative partnerships, stakeholders can streamline processes, reduce timelines, and improve data quality. Attendees will gain insights into best practices for fostering collaboration, harnessing technology, and overcoming common challenges to accelerate clinical trial success and bring therapies to patients faster.
14:45 Innovative Strategies in Gene Therapy Clinical Trial Design to Enhance Operational Efficiency and Data Integrity
This session will cover cutting-edge approaches to designing gene therapy clinical trials that optimize operational workflows while ensuring robust data integrity. Attendees will gain insights into novel trial frameworks, adaptive methodologies, and digital tools that streamline study execution and improve data accuracy. Emphasis will be placed on overcoming unique challenges in gene therapy trials, such as patient recruitment, complex endpoints, and regulatory compliance. This session aims to equip researchers, clinicians, and trial managers with practical strategies to accelerate development timelines without compromising scientific accuracy.
15:00 Interactive Discussion
15:30 Refreshment Break
CLINICAL & REGULATORY CHALLENGES PANEL DISCUSSION
16:00 Maximizing Success in Cell and Gene Trials through Eligibility Precision, Adaptive Design, Monitoring Innovation, and Analytics (15 minutes each section)
16:00 Navigating Patient Eligibility & HLA Matching in Cell
and Gene Therapies: Overcoming Immune and Disease Stage Challenges to Ensure Optimal Treatment Success
16:15 Overcoming Clinical Trial Design Challenges in Rare Disease Populations: Innovative Approaches to Endpoint Definition, Statistical Significance, and Regulatory Standards in Small Patient Cohorts
16:30 Long-Term Follow-Up in Cell and Gene Therapy: Addressing Safety, Efficacy, and High Costs in Lifelong Monitoring for Durable Treatment Outcomes
16:45 Regulatory Pathways for Advanced Therapies: Navigating Evolving Frameworks, Regulatory Uncertainty, and Approval Timelines for Emerging Technologies like CRISPR and Base Editing.
17:00 The Search for Reliable Biomarkers in Cell and Gene Therapies: Developing Validated Biomarkers to Improve Diagnosis, Monitoring, and Clinical Efficacy Measurement
17:15 Panelist:
Hamzeh Migdadi Medical Director, CNS Gene Therapy Eli Lilly and Company
Adam Mendizabal Senior Vice President, Global Head of Cell and Gene Therapy Emmes
17:45 End of day one