CASSS Sharing Science Solutions CGTP 2025

Description

+ Include:  videos + file sub vtt +  pdf, size:  GB

+ Target Audience: Medical Geneticists, Hematologists, Oncologists, and Immunologists

+ Sample video: contact me for sample video

+ Information:

The CASSS Cell and Gene Therapy Products (CGTP) 2025 program provides an in-depth exploration of the latest advancements, analytical challenges, and regulatory frameworks in the rapidly evolving field of cell and gene therapies. The symposium covers critical aspects of product lifecycle management, including chemistry, manufacturing, and controls (CMC), potency assay development, genome editing innovations (such as CRISPR and Prime Editing), viral vector characterization, and global regulatory strategies.

Learning Objectives

• Implement advanced analytical and sequencing approaches for comprehensive viral vector (AAV) characterization and process-related impurity tracking.

• Navigate CMC challenges to develop robust potency assays for cell and gene therapies across different phases of the product lifecycle.

• Evaluate strategies for ensuring product comparability during multifactorial changes in cell therapy manufacturing.

• Assess the clearance and clinical effects of genome-editing components, particularly for ex-vivo allogeneic cell therapy products.

• Apply global regulatory insights and ICH criteria to streamline product release, manage exceptional release bottlenecks, and meet donor selection standards.

• Optimize analytical release testing methodologies by utilizing mass photometry, multiplex platforms, and rapid sterility methods.

Target Audience

• This program is designed for scientists, engineers, quality control professionals, and regulatory affairs specialists involved in the development, manufacturing, and commercialization of cell, gene, and tissue-based therapies.
• Best for Medical Geneticists, Hematologists, Oncologists, and Immunologists.

Topics

• A Matrix Approach to Characterize a Stem Cell-Derived Cell Product During Early Development

• A Single, Ready-to-Use Kit for Fast, Comprehensive Analysis of Both Plasmid Topological Isoforms and Linear Sizing on a Multi-Capillary Electrophoresis…

• Accelerating Access to Life-Saving Cell Therapies Leveraging Platform Technologies

• Advanced Sequencing Approaches for Comprehensive AAV Vector Characterization

• Advancing Analytical Development for Prime Editing Therapeutics A Platform-Based Approach

• Advancing Genomic Medicine Navigating Challenges in CMC Potency Assay Development Throughout the Product Life-Cycle

• Analytics for Process-related Impurities in Viral Vector Manufacturing Presented by Cygnus Technologies LLC

• Anticipating the Exceptional Release Bottleneck

• ATMP Activities at MEB

• Beyond “One Disease at a Time” Genetic Therapy Platforms for Rare Monogenic Disease

• Breakfast Chat FDA START Pilot Program A Participant Perspective

• CASSS Welcome CGTP 2025 Introduction / CASSS Welcome CGTP Summit 2025 Introduction

• Challenges in Getting Genome Editing Medicines into the Clinic

• Closing Remarks and Invitation to CGTP 2026 / Closing Remarks Invitation to CGTP Summit 2026

• Considerations in Development Characterization and Commercialization of Platform Processes

• Cost Drivers

• CRISPR Genome Editing Components Used for Ex-Vivo Genome-Editing of Allogeneic Cell Therapy Products Their Clearance and Their Effects

• Developing AAV-Based Gene Therapies Amidst Some Real Concerns and Some empty Threats

• Developing and Deploying N-of-1 Gene-Editing Therapies

• Donor Selection Criteria Across ICH Regions

• Ensuring Product Comparability in Cell Therapy Approach to Managing Multifactorial Changes

• Flash Poster Talks – Session 1 & 2

• From Complexity to Simplicity Phase-Appropriate Development of a QC- Friendly Gene Therapy Potency Assay

• Global Health in Cell and Gene Therapy

• Healthy Donor Cells Compared to SCD and TDT Patient Cells in Casgevy Process Performance and Product Quality

• INS1203 RNA-End Joining Technology Enables A Dual AAV Approach for ABCA4 Gene Replacement in Stargardt Disease

• Integrating Process and Analytics A Prerequisite to Streamlining the Production of Viral Vectors

• Introduction to CASGEVY The First CRISPR-Cas9 Based Commercially Approved Therapy For SCD and TDT

• Investigating the Aggregation Genome Release and Self-Interactions of Adeno-Associated Virus Formulations

• Is the Vehicle Empty or Full Mass Photometry for AAV Capsid EF Assessment in the GMP Space

• Keynote Presentation Developing and Deploying N-of-1 Gene-Editing Therapies

• Lipid Nanoparticles-Points to Consider For Non-Viral Delivery of RNA-based Therapeutics

• Multiplex Approaches to Analytical Platforms for CGT Release

• Non-Traditional Approaches to Comparability

• Optimizing Analytical Release Testing Through Reduced Volume and Turn-around Times

• Parallel Session 3 – Advancing Stem Cell Therapy Development Overcoming Challenges and Expanding Horizons

• Parallel Session 4 – Development and Characterization of Viral Vectors

• Parallel Session 7 – Optimizing the Analytical Testing Panel

• Parallel Session 8 – Non-Traditional Modes of Delivery for Ex-vivo and In-vivo Gene Therapies

• Phase Appropriate Engineering Run Approaches and Stability

• Plenary Session 1 – Gene Editing

• Plenary Session 2 – Fireside Chat Non-Profit Alliance Updates

• Plenary Session 5 – Platform Development

• Plenary Session 6 – Fireside Chat Investing in CGT

• Plenary Session 9 – Global Regulatory Panel

• Potency Assurance Strategies for Cell and Gene Therapies

• Recent Regulatory Advancements on Cell and Gene Therapy Products in Japan

• Reducing COGM in Cell Gene Therapy – The CDMO Perspective Sponsored Lunch Session

• Session I – Building Strong Foundations Early Phase Strategies for Potency Assays

• Session II – Challenges and Opportunities for Potency Assays

• Session III – Deep Dives into Key Topics in Cell and Gene Therapy Potency

• Session IV – Navigating Potency Assays in Cell and Gene Therapy Global Regulatory Perspectives

• Setting Specifications for Autologous CAR-T Cell Products

• Strategy for Potency Determination of Gene Therapy Products – Overview of the BioPhorum GT Potency Strategy Workstream

• Transparency-Driven Partnerships for Shared Success – iPSC Journey

• Use of New Rapid Sterility Testing Methods – Successes and Challenges