CASSS Sharing Science Solutions CGTP 2025

Cell & Gene Therapy • Gene Editing • AAV & Viral Vectors • CMC • Potency • Analytical Development • Regulatory Science

Explore the evolving science, manufacturing strategies, analytical technologies, and regulatory considerations shaping advanced therapies with CASSS Sharing Science Solutions CGTP 2025, the Cell and Gene Therapy Products (CGTP) Symposium: Manufacturing, Quality and Regulatory Considerations.

Organized by CASSS – Sharing Science Solutions, CGTP 2025 was held June 10–12, 2025, at the Bethesda North Marriott Hotel & Conference Center in Rockville, Maryland, USA. The symposium brought together professionals from industry, regulatory agencies, and academia to discuss scientific and technical challenges affecting the development, manufacturing, characterization, quality control, and regulation of cell and gene therapy products.

The program addressed major areas including viral-vector characterization, AAV analytics, genome editing, CRISPR, Prime Editing, potency strategies, product comparability, analytical release testing, manufacturing and CMC challenges, process-related impurities, donor selection, global regulatory requirements, and next-generation cell and gene therapy development.

Course Details

• Program: Cell and Gene Therapy Products – CGTP 2025
• Organizer: CASSS – Sharing Science Solutions
• Full Symposium Title: Cell and Gene Therapy Products: Manufacturing, Quality and Regulatory Considerations
• Year: 2025
• Dates: June 10–12, 2025
• Venue: Bethesda North Marriott Hotel & Conference Center
• Location: Rockville, Maryland, USA
• Format: In-Person and Virtual Participation
• Primary Field: Cell and Gene Therapy
• Scientific Focus: Manufacturing, Quality, Analytics, CMC and Regulatory Science
• Language: English

A separate CGTP Summit was held on June 9, 2025, immediately before the symposium. The Summit focused particularly on CMC-related delays in clinical development and strategies for developing globally acceptable potency approaches for cell and gene therapy products.

Course Overview

Cell and gene therapies introduce analytical, manufacturing, and regulatory challenges that differ substantially from those encountered with many conventional pharmaceutical products.

Development may require integration of:

Product Design → Manufacturing Process → Analytical Characterization → Potency → Comparability → Release Testing → Regulatory Strategy → Clinical Development

CASSS CGTP 2025 provides a multidisciplinary forum for examining these interconnected issues.

The symposium focuses on the scientific and regulatory questions involved in bringing complex products from early development toward clinical use and commercialization.

Major areas of discussion include:

• Cell therapy products
• Gene therapy products
• AAV vectors
• Viral-vector manufacturing
• Genome editing
• CRISPR-based therapies
• Prime Editing
• Potency assays
• Product comparability
• Product characterization
• Process-related impurities
• Analytical platform technologies
• Release testing
• Sterility testing
• CMC strategy
• Regulatory science
• Global regulatory harmonization
• Donor selection
• CAR-T products
• Product lifecycle management

Learning Objectives

Upon completion of this program, participants should be better able to:

• 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.

CGTP 2025 Scientific Program

The scientific program combines:

• Plenary sessions
• Parallel scientific tracks
• Regulatory discussions
• Speaker presentations
• Panel discussions
• Roundtable discussions
• Technical seminars
• Poster presentations
• Scientific networking

CASSS describes the symposium as a forum for exchanging scientific ideas and maintaining dialogue with regulators as regulatory practices continue to evolve for complex cell and gene therapy products.

Viral Vector & AAV Characterization

Adeno-associated virus (AAV) remains an important delivery platform in gene therapy, making comprehensive vector characterization a major analytical priority.

CGTP 2025 included advanced discussion of sequencing approaches for AAV characterization.

The available CASSS scientific material demonstrates how next-generation sequencing (NGS) can complement established AAV analytical tools.

Relevant analytical areas include:

• Vector identity
• Packaged nucleic-acid content
• Sequence contaminants
• Genome composition
• Vector genome titer
• Capsid characterization
• Product-related impurities
• Process-related impurities

Short-read sequencing can support vector identity analysis, while long-read sequencing can provide additional information about the composition of packaged DNA molecules.

A comprehensive analytical strategy may integrate:

Sequencing + Molecular Testing + Physical Characterization + Impurity Analysis + Functional Testing

Viral Vector Manufacturing & Process-Related Impurities

Viral-vector manufacturing requires close coordination between process development and analytical characterization.

CGTP 2025 included dedicated discussion on:

Analytics for Process-related Impurities in Viral Vector Manufacturing

as well as broader consideration of integrating process and analytical development for viral-vector production.

Important considerations include:

• Manufacturing consistency
• Process understanding
• Residual materials
• Product-related impurities
• Process-related impurities
• Analytical sensitivity
• Method suitability
• Lifecycle control

Genome Editing

Genome editing was a major scientific theme of CGTP 2025.

The program examined the challenges involved in translating emerging genome-editing technologies from research into clinical development.

A dedicated scientific session addressed:

Challenges in Getting Genome Editing Medicines into the Clinic

with discussion extending across scientific, analytical, manufacturing, and regulatory issues.

Relevant areas include:

• Editing efficiency
• Product quality
• Off-target considerations
• Process control
• Impurity characterization
• Analytical testing
• Clinical translation
• CMC development
• Regulatory expectations

CRISPR Genome Editing

CGTP 2025 included specific analysis of genome-editing components used in ex-vivo allogeneic cell therapy products.

The presentation:

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

examined the fate and potential effects of editing components used during manufacturing.

This area is important because genome editing introduces additional materials and process steps that may need to be characterized and controlled before product release.

A relevant framework is:

Editing Components → Manufacturing Process → Clearance Assessment → Residual Testing → Product Characterization

Prime Editing

Next-generation genome-editing approaches were also represented within the official CGTP 2025 program.

A dedicated presentation addressed:

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

as part of the genome-editing scientific session.

Prime Editing illustrates how rapidly evolving therapeutic platforms may require new analytical strategies capable of supporting:

• Product characterization
• Process development
• Potency assessment
• Impurity monitoring
• Quality control
• Clinical development

N-of-1 Gene-Editing Therapies

One of the prominent scientific themes at CGTP 2025 was the development of highly individualized gene-editing therapies.

The symposium featured:

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

presented by Kiran Musunuru, University of Pennsylvania.

CASSS later highlighted this presentation when summarizing the 2025 meeting and identified individualized gene therapy as an example of collaboration between academia, industry, regulators, and clinical institutions.

Potency Assay Development

Potency is one of the most important and challenging quality attributes for many cell and gene therapy products.

The broader CGTP 2025 program included extensive discussion of potency assurance strategies, including dedicated roundtables.

Potency development may require consideration of:

• Mechanism of action
• Product complexity
• Biological variability
• Development phase
• Assay sensitivity
• Assay precision
• Reference standards
• Manufacturing changes
• Product comparability

A lifecycle-oriented strategy can be summarized as:

Understand Mechanism → Identify Relevant Biological Activity → Develop Assay → Establish Control Strategy → Refine During Development

CGTP Summit 2025 – Potency Strategy

The preceding CGTP Summit 2025 focused extensively on developing globally acceptable potency strategies.

Regulators from multiple regions discussed challenges surrounding:

• Potency-assay development
• Potency matrices
• Biological assays
• Alternative assay readouts
• Reference-standard bridging
• Manufacturing consistency
• Comparability
• Global regulatory harmonization

The program brought together regulatory perspectives from organizations including European, Canadian, and Japanese authorities.

Product Comparability

Manufacturing changes are common throughout the lifecycle of advanced therapy products.

Changes may involve:

• Raw materials
• Manufacturing sites
• Equipment
• Processes
• Scale
• Analytical methods
• Formulation
• Product specifications

CGTP 2025 included the presentation:

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

along with roundtables examining both traditional and non-traditional approaches to comparability.

A comparability strategy aims to establish whether manufacturing changes alter clinically meaningful product attributes.

Cell Therapy Manufacturing

Cell therapy manufacturing involves inherent biological complexity and variability.

Important considerations can include:

• Starting material
• Donor characteristics
• Cell phenotype
• Manufacturing consistency
• Process controls
• Potency
• Product specifications
• Comparability
• Sterility
• Release testing

The CGTP 2025 program also included discussion of specifications for autologous CAR-T cell products, particularly in the context of patient-to-patient variability in starting material.

Donor Selection

For cell-derived therapies, donor selection may influence product quality, regulatory compliance, and patient safety.

A dedicated CGTP 2025 roundtable addressed:

Donor Selection Criteria Across ICH Regions for Cells Used as Starting Material for ATMP Manufacture

highlighting the need to understand differences and similarities among international regulatory approaches.

Analytical Release Testing

Reliable release testing is essential before advanced therapy products can progress to clinical or commercial use.

CGTP 2025 explored strategies for improving analytical efficiency while maintaining appropriate quality control.

Topics included:

• Potency testing
• Product identity
• Purity
• Sterility
• Analytical platform development
• Multiplex assays
• Product specifications
• Rapid testing
• Release strategies

Multiplex Analytical Platforms

The official CGTP 2025 roundtable program included:

Multiplex Approaches to Analytical Platforms for CGT Release

reflecting interest in methods capable of evaluating multiple analytical characteristics while improving efficiency and reducing testing burden.

Multiplex approaches may support:

Fewer Individual Tests → More Integrated Data → Faster Analytical Workflows → Improved Release Efficiency

while still requiring appropriate validation and regulatory justification.

Rapid Sterility Testing

Traditional sterility-testing timelines can create particular challenges for some cell therapies because products may have:

• Short shelf lives
• Time-sensitive administration
• Limited manufacturing windows
• Patient-specific production processes

The official CGTP 2025 roundtable program included:

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

This reflects continued efforts to develop microbiological testing approaches compatible with the unique timelines of advanced therapy products.

CAR-T Cell Products

CAR-T therapies combine biological variability with complex manufacturing and analytical requirements.

CGTP 2025 addressed issues including:

• Starting-material variability
• Product specifications
• Potency
• Manufacturing consistency
• Analytical testing
• Comparability
• Release criteria

A dedicated roundtable examined:

Setting Specifications for Autologous CAR-T Cell Products Given the Inherent Patient to Patient Variability of the Apheresis Starting Material.

CMC Challenges in Cell & Gene Therapy

Chemistry, Manufacturing, and Controls (CMC) form a central theme across the CGTP program.

CMC strategies must evolve as products move through:

Research → Early Clinical Development → Late Clinical Development → Registration → Commercialization

Important considerations include:

• Phase-appropriate assays
• Process development
• Potency
• Analytical method development
• Manufacturing changes
• Product comparability
• Stability
• Product specifications
• Release testing
• Supply chain
• Regulatory expectations

The CGTP Summit specifically focused on CMC-related delays affecting clinical development.

Global Regulatory Strategies

Cell and gene therapy development increasingly occurs across multiple regulatory jurisdictions.

CGTP 2025 concluded with a Global Regulatory Panel featuring regulatory leaders representing agencies and perspectives from multiple regions, including Europe, Canada, and Japan.

The discussion focused on how regulators can continue supporting increasingly diverse and complex products while maintaining appropriate standards for:

• Quality
• Safety
• Effectiveness
• Manufacturing control
• Clinical development
• Timely patient access

Global Regulatory Harmonization

International development programs may encounter differences in:

• Analytical requirements
• Donor-selection criteria
• Product specifications
• Manufacturing expectations
• Release criteria
• Potency strategies
• Regulatory documentation

The symposium provides an important forum for dialogue between developers and regulators regarding these issues.

The broader goal can be summarized as:

Scientific Understanding + Regulatory Dialogue + Global Alignment → More Efficient CGT Development

Global Health & Access to Cell and Gene Therapy

The official roundtable program also included discussion of:

Global Health Equity in Cell and Gene Therapy

highlighting challenges surrounding access and sustainable development of advanced therapies beyond a small number of healthcare systems.

CASSS identified sustainability in cell and gene therapy development and commercialization as one of the notable themes emerging from the 2025 meeting.

Scientific Collaboration

CGTP is designed around interaction between:

Industry + Academia + Regulators + Scientists + Engineers + Quality Professionals

CASSS emphasizes scientific exchange and dialogue with regulators as a core purpose of the symposium.

The 2025 meeting included professionals from 17 countries, with participation from 104 companies and 11 regulators, demonstrating the international and cross-sector nature of the event.

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.

It is particularly relevant for:

• Medical Geneticists
• Hematologists
• Oncologists
• Immunologists
• Gene Therapy Researchers
• Cell Therapy Scientists
• Molecular Biologists
• Clinical Researchers
• Analytical Scientists
• Process Development Scientists
• CMC Professionals
• Quality Control Professionals
• Quality Assurance Professionals
• Regulatory Affairs Specialists
• Biopharmaceutical Scientists
• Manufacturing Scientists and Engineers
• Professionals involved in CAR-T and other advanced cellular therapies

Who Should Take This Course?

CASSS Sharing Science Solutions CGTP 2025 is particularly relevant for professionals working at the interface of advanced therapeutic science, manufacturing, analytics, quality, and regulation.

The program is suited to professionals seeking updates in:

• Cell therapy
• Gene therapy
• Medical genetics
• Genome editing
• CRISPR
• Prime Editing
• AAV vector development
• Viral-vector characterization
• CAR-T therapy
• Potency assays
• Product comparability
• Analytical method development
• Release testing
• Manufacturing and CMC
• Quality control
• Regulatory science

Why This Course Is Useful

The strength of CGTP 2025 is its focus on the complete development pathway rather than a single analytical or clinical topic.

The program connects:

Gene & Cell Therapy Science → Manufacturing → Analytics → Potency → Comparability → Quality → Regulatory Strategy → Clinical Translation

It also brings together industry, academic, and regulatory perspectives, allowing participants to examine how scientific innovations translate into practical development and regulatory strategies.

CGTP 2025 By the Numbers

According to CASSS’s official 2025 summary:

• 203 attendees participated in person
• 126 attendees participated online
• 144 were first-time attendees
• 11 regulators participated
• 104 companies were represented
• 17 countries were represented

Participating countries included the United States, Canada, United Kingdom, several European nations, China, Japan, South Korea, Taiwan, and Saudi Arabia.

Major Topics Covered

• Cell and Gene Therapy Products
• Advanced Therapy Medicinal Products – ATMPs
• Gene Therapy
• Cell Therapy
• Genome Editing
• CRISPR Genome Editing
• Prime Editing
• N-of-1 Gene-Editing Therapies
• AAV Gene Therapy
• Viral Vector Characterization
• Advanced Sequencing
• Short-Read Sequencing
• Long-Read Sequencing
• Process-Related Impurities
• Viral Vector Manufacturing
• CMC Strategy
• Potency Assays
• Potency Assurance
• Product Comparability
• Multifactorial Manufacturing Changes
• CAR-T Cell Products
• Autologous Cell Therapy
• Allogeneic Cell Therapy
• Donor Selection
• Analytical Development
• Analytical Release Testing
• Multiplex Analytical Platforms
• Rapid Sterility Testing
• Product Specifications
• Manufacturing Control
• Regulatory Science
• Global Regulatory Strategy
• ICH Considerations
• Global Regulatory Harmonization
• Global Health Equity
• CGT Commercialization
• Clinical Translation

Short Description

CASSS Sharing Science Solutions CGTP 2025 is the Cell and Gene Therapy Products: Manufacturing, Quality and Regulatory Considerations symposium held June 10–12, 2025, at the Bethesda North Marriott Hotel & Conference Center in Rockville, Maryland.

The program explores cell and gene therapy development, AAV and viral-vector characterization, advanced sequencing, CRISPR and Prime Editing, potency assays, product comparability, CAR-T manufacturing, CMC strategy, analytical release testing, rapid sterility testing, donor selection, and global regulatory considerations.

Organizer: CASSS – Sharing Science Solutions
Dates: June 10–12, 2025
Venue: Bethesda North Marriott Hotel & Conference Center
Location: Rockville, Maryland, USA
Format: In-Person and Virtual
Primary Focus: Cell & Gene Therapy Manufacturing, Quality, Analytics and Regulatory Science

Topics

• Advanced AAV Characterization: Sequencing and analytical strategies for vector identity, packaged nucleic acids, impurities, and product characterization.
• Viral Vector Manufacturing: Integration of process development and analytics with monitoring of process-related impurities.
• Genome Editing: Scientific, manufacturing, analytical, and regulatory challenges involved in translating gene-editing technologies into clinical products.
• CRISPR: Clearance and effects of genome-editing components used in ex-vivo allogeneic cell therapy manufacturing.
• Prime Editing: Platform-based analytical development strategies for next-generation gene-editing therapeutics.
• N-of-1 Gene Editing: Development and deployment of individualized gene-editing therapeutic approaches.
• Potency Assurance: Development and lifecycle management of potency strategies for complex cell and gene therapy products.
• Product Comparability: Strategies for establishing comparability following multifactorial manufacturing changes.
• CAR-T Products: Specifications and analytical control for autologous cellular products with variable patient-derived starting materials.
• Donor Selection: Donor-selection criteria across ICH regions for cells used as ATMP starting materials.
• Analytical Release Testing: Strategies for efficient and scientifically appropriate product-release testing.
• Multiplex Platforms: Multiplex analytical approaches designed to improve CGT release-testing efficiency.
• Rapid Sterility Testing: New microbiological testing approaches and implementation challenges.
• CMC Strategy: Manufacturing, control, potency, analytical, stability, and development challenges across the product lifecycle.
• Global Regulatory Strategy: Regulatory expectations and evolving approaches across major global health authorities.
• Global Health Equity: Challenges associated with expanding access to cell and gene therapies internationally.

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