Innovative Idea Generating Centre
CELL & GENE THERAPY SUMMIT 2025
Redefining Possibilities Through Smarter Delivery, Safer Editing, and Scalable Innovation
DATE: 12th November , 2025 2025(Time Zone CT) | Virtual Congress
08:50 Chairperson Opening Remarks
BREAKING BIOLOGICAL BARRIERS
09:00 When Rare Isn’t So Rare - Broadening The Scope Of Rare Disease Targeting
Jennifer Johnston CEO NysnoBio
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 Interactive Panel Discussion: Advancing CRISPR Precision
for Safer Genetic Engineering
Panelist:
- Brad Ringeisen Executive Director Innovative Genomics Institute
- Xiaoya Sun Scientist, Analytical Development, Cell Therapy Takeda
11:45 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
FOCUSED ULTRASOUND
14:00 A Non-Invasive Delivery Platform for Gene and Cell Therapy to the Brain—and Beyond
- Overview of focused ultrasound (FUS) as a modality to non-invasively and transiently open the blood-brain barrier (BBB) for therapeutic delivery —including clinical data
- Recent advances in preclinical studies using FUS to deliver gene and cell therapies to the brain in small and large animal models
- Toward first-in-human translation: ongoing efforts to advance FUS-mediated delivery of gene therapy to the clinic
- Emerging preclinical efforts expanding FUS-mediated gene therapy delivery to large organs (e.g., liver, heart) Challenges and opportunities for clinical translation: a framework in development to guide indication selection and align with regulatory, manufacturing, and access considerations
Frederic Padilla Director Gene and Cell Therapy Program Focused Ultrasound Foundation
14:25 Questions & Discussion
14:30 Driving Clinical Research Forward: Enhancing Awareness, Diversity, and Execution with Digital Innovation and Strategic Service Integration.
This session explores how Clinical Central Services leverages digital innovation and emerging technologies to optimize research accessibility, enhance diversity, reduce operational burden, and accelerate clinical trial execution. Learn how strategic service integration and third-party coordination drive excellence in capability design and delivery across the clinical research ecosystem.
14:55 Questions & Discussion
15:00 Refreshment Break
CLINICAL & REGULATORY CHALLENGES PANEL DISCUSSION
15:30 Maximizing Success in Cell and Gene Trials through Eligibility Precision, Adaptive Design, Monitoring Innovation, and Analytics (15 minutes each section)
15:30 Navigating Patient Eligibility & HLA Matching in Cell
and Gene Therapies: Overcoming Immune and Disease Stage Challenges to Ensure Optimal Treatment Success
15:45 Overcoming Clinical Trial Design Challenges in Rare Disease Populations: Innovative Approaches to Endpoint Definition, Statistical Significance, and Regulatory Standards in Small Patient Cohorts
16:00 Long-Term Follow-Up in Cell and Gene Therapy: Addressing Safety, Efficacy, and High Costs in Lifelong Monitoring for Durable Treatment Outcomes
16:15 Regulatory Pathways for Advanced Therapies: Navigating Evolving Frameworks, Regulatory Uncertainty, and Approval Timelines for Emerging Technologies like CRISPR and Base Editing.
16:30 The Search for Reliable Biomarkers in Cell and Gene Therapies: Developing Validated Biomarkers to Improve Diagnosis, Monitoring, and Clinical Efficacy Measurement
17:15 Interactive Panel Discussion: Optimizing CGT Clinical Development: Navigating Eligibility, Rare Populations, Lifelong Monitoring, and Digital Analytics
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