CELL & GENE THERAPY SUMMIT 2025

ADVANCING GENE THERAPIES WITH SMARTER VECTORS, IMPROVED CRISPR PRECISION, AND SCALABLE MANUFACTURING
DATE: 11TH JULY 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.

09:55 Questions & Discussion
 

ADVANCING CRISPR-CAS9 PRECISION PANEL DISCUSSION
10:00 CRISPR Under the Microscope: Innovations for Precision and Safety in Genome Editing

  • 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. 

  • Off-Target Effects in CRISPR: A Critical Barrier to Reliable Therapeutic and Agricultural Applications. 

This talk explores the significance of off-target effects in CRISPR technology, discussing their impact on both therapeutic interventions and agricultural advancements. We’ll examine current challenges, potential risks, and strategies for improving precision. 

  • 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. 

  • 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. 

 

  • 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 Questions & Discussion 

 

11:30 Refreshment Break

 

PANEL DISCUSSION ON 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

INNOVATIONS IN TREATMENT DURABILITY PANEL DISCUSSION
14:00 Strategies For Prolonged Response Without Frequent Re-Administration Of Therapy (15 minutes each section)

  • Revolutionizing Disease Treatment- The Impact of
    Cell and Gene Therapies on Genetic and Acquired
    Disorders: This presentation explores the ground
    breaking potential of cell and gene therapies in
    transforming the treatment of both genetic and
    acquired disorders. By directly targeting the
    underlying genetic and molecular causes of these
    diseases, these innovative therapies offer a new era of
    hope for patients facing conditions once considered
    untreatable. 
  • Challenge of Durability: This session will explore the
    promising early outcomes of cell and gene therapies,
    while addressing the ongoing uncertainty surrounding
    their long-term effectiveness 
  • The Impact of Cell and Gene Therapies on Genetic
    and Acquired Disorders: This presentation delves into
    the revolutionary potential of cell and gene therapies
    in reshaping the treatment of genetic and acquired
    disorders. These therapies work by precisely targeting
    the genetic and molecular foundations of these
    diseases.
  • Overcoming Biological, Technological, and Clinical
    Barriers: Enhancing Durability of Gene Therapies
    through Vector Design, Immunomodulation, and
    Regenerative Medicine: This talk explores strategies to
    improve the durability of gene therapies by
    addressing biological, technological, and clinical
    challenges. It covers innovative vector designs for
    more efficient delivery, the role of immunomodulation
    in reducing immune responses, and advancements in
    regenerative medicine to support long-term
    therapeutic outcomes. These integrated approaches
    aim to revolutionize gene therapy success.
  • Future Outlook: Ensuring Long-Term Success in Cell &
    Gene Therapies: Achieving durable efficacy in cell and
    gene therapies demands that we address key
    challenges head-on. This session explores the critical
    steps needed to secure sustained benefits for patients
    and maximize the transformative potential of these
    innovative treatments in real-world clinical settings.

 

15:15 Questions & Discussion
 

15:30 Refreshment Break
CLINICAL & REGULATORY CHALLENGES PANEL DISCUSSION
16:00 Optimizing Patient Eligibility, HLA Matching, And Overcoming Challenges In Cell And Gene Therapies: Innovations In Clinical Trials, Biomarkers, And Regulatory Pathways For Long Term Success (15 minutes each section)

  • Navigating Patient Eligibility & HLA Matching in Cell
    and Gene Therapies: Overcoming Immune and Disease Stage Challenges to Ensure Optimal Treatment Success
  • Overcoming Clinical Trial Design Challenges in Rare Disease Populations: Innovative Approaches to Endpoint Definition, Statistical Significance, and Regulatory Standards in Small Patient Cohorts
  • Long-Term Follow-Up in Cell and Gene Therapy: Addressing Safety, Efficacy, and High Costs in Lifelong Monitoring for Durable Treatment Outcomes
  • Regulatory Pathways for Advanced Therapies: Navigating Evolving Frameworks, Regulatory Uncertainty, and Approval Timelines for Emerging Technologies like CRISPR and Base Editing. 
  • The Search for Reliable Biomarkers in Cell and Gene Therapies: Developing Validated Biomarkers to Improve Diagnosis, Monitoring, and Clinical Efficacy Measurement 

 

17:15 Questions & Discussion 

 

REIMBURSEMENT MODELS

 17:30 Addressing The High Cost Of Cell And Gene Therapies: Reimbursement Models And Global Access Challenges 

 

This talk examines the rising costs of cell and gene therapies, focusing on the financial barriers faced by patients and healthcare systems. It delves into innovative reimbursement models and strategies aimed at improving affordability. Additionally, it addresses the challenges of ensuring global access to these ground-breaking therapies, particularly in low-resource settings. 

 

17:55 Questions & Discussion 

 

ENGINEERING THE CURE 

18:00 Navigating Manufacturing Precision In Ex Vivo Therapies Versus Delivery Barriers In In Vivo Gene Medicine 

 

This session offers an in-depth look at the contrasting engineering challenges in gene medicine: achieving manufacturing precision for ex vivo therapies versus overcoming biological delivery barriers in in vivo approaches. It highlights innovations in scalable production, vector design, and targeted delivery, offering insights into how these technologies are shaping the future of personalized and regenerative medicine. 

 

18:25 Questions & Discussion 

 

18:30 End of conference

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