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Cell and Gene Therapy CMC and Regulatory Strategy Course & Certification
Intermediate
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3 hours
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Cell and Gene Therapy CMC and Regulatory Strategy
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About
Cell and gene therapies present unique manufacturing, quality, and regulatory challenges due to their complex biological nature, limited starting materials, and highly controlled production processes. Developing an effective Chemistry, Manufacturing and Controls (CMC) strategy is essential for demonstrating product quality, safety, consistency, and regulatory compliance throughout the development and commercial lifecycle of Advanced Therapy Medicinal Products (ATMPs).
This GMP – Cell and Gene Therapy CMC and Regulatory Strategy Training Course & Certification provides comprehensive knowledge of global regulatory frameworks, CMC development strategies, GMP requirements, raw material control, manufacturing process design, analytical methods, potency testing, comparability assessments, technology transfer, process validation, stability programmes, supply chain management, regulatory submissions, lifecycle management, and quality risk management for cell and gene therapy products. Upon successful completion, learners receive a certification demonstrating their understanding of CMC strategy, GMP compliance, and regulatory expectations for cell and gene therapy development and manufacturing.
Who Should Enrol?
- Cell and Gene Therapy Manufacturing Professionals
- CMC Development and Process Development Scientists
- Quality Assurance and Quality Management Professionals
- Regulatory Affairs and CMC Regulatory Professionals
- Analytical Development and Quality Control Scientists
- Technology Transfer and Validation Engineers
- Biopharmaceutical Manufacturing and Operations Personnel
- Anyone involved in the development, manufacture, quality, or regulatory management of cell and gene therapy products
What you will learn
Understand the regulatory framework for cell and gene therapies, CMC expectations, product classification, and the unique challenges associated with Advanced Therapy Medicinal Products (ATMPs).
Learn how to develop robust CMC strategies covering raw materials, manufacturing processes, control strategies, analytical methods, comparability, and product lifecycle management.
Develop knowledge of GMP manufacturing, process validation, potency assays, stability programmes, technology transfer, supply chain controls, and regulatory documentation for cell and gene therapy products.
Gain practical understanding of regulatory submissions, quality risk management, post-approval changes, global regulatory expectations, and best practices for maintaining compliance throughout the product lifecycle.
Course Syllabus
- Autologous and allogeneic cell therapies
- CAR-T, TCR-T, NK-cell and stem-cell products
- In vivo and ex vivo gene therapies
- Viral and non-viral gene delivery
- Gene editing and genome engineering
- Tissue-engineered products and combined ATMPs
- US biological product and IND/BLA framework
- FDA CBER Office of Therapeutic Products
- IMPD, CTA and marketing-authorisation pathways
- UK ATMP and clinical-trial environment
- Hospital exemption and its limitations
- Orphan, expedited and rare-disease pathways
- Product classification and designation
- Early scientific advice and INTERACT-type meetings
- Pre-IND, Type B and Type C interactions
- Global development and regional divergence
- What is a CMC target product profile?
- Intended use, patient population and route of administration
- Dose, presentation and administration model
- Autologous turnaround time and allogeneic batch scale
- Shelf life, storage, transport and point-of-care handling
- Critical quality attributes and product-performance expectations
- Phase-appropriate strategy across development stages
- Progressive control strategy and risk-based specifications
- Platform knowledge and prior knowledge
- FDA 2026 CMC flexibilities for CGT BLA development
- When flexibility does not remove the need for scientific justification
- The integrated CMC development plan
- CMC risk register
- Critical path and long-lead items
- Manufacturing-site strategy
- Analytical-development, potency-assurance and comparability/change-management plans
- CMC target product profile workshop
- Patient- or donor-derived cells and cellular starting materials
- Master and working cell banks
- Plasmids and nucleic-acid templates
- Viral vector intermediates and gene-editing reagents
- Serum, cytokines, media and growth factors
- Single-use assemblies, enzymes, beads and processing reagents
- Human- and animal-derived material risks
- Pharmacopoeial versus non-pharmacopoeial materials
- Supplier qualification and change notification
- Chain of identity
- Chain of custody
- Collection-site controls: apheresis and tissue collection
- Labelling, reconciliation and temperature/transit monitoring
- Mix-up prevention and electronic tracking
- Autologous chain-of-identity simulation
- Donor eligibility and testing
- Microbial control strategy, mycoplasma and adventitious-agent control
- Viral safety principles and replication-competent virus risk
- Starting-material risk assessment exercise
- Cell selection, enrichment, activation and modification
- Expansion, harvest, washing, formulation and cryopreservation
- Closed and functionally closed processing
- Autologous scheduling and vein-to-vein time
- Allogeneic scale-up and scale-out
- Vector production platforms - a strategic view
- Transfection, infection, harvest and downstream processing
- Critical quality attributes, critical material attributes and critical process parameters
- In-process controls and real-time information
- Quality by Design and design space
- Segregation and campaign manufacture
- Unidirectional flows, closed systems and isolators
- Environmental monitoring, aseptic processing and contamination control strategy
- Biosafety and genetically modified organism controls
- Control strategy: material, process and analytical controls
- Release, shipping and administration controls
- CQA-CPP mapping workshop
- Autologous versus allogeneic control comparison
- Method purpose and lifecycle
- Fit-for-purpose versus validated methods
- Orthogonal methods and reference standards
- Sampling and limited-volume challenges
- Product testing: identity, viability and cell count
- Purity, impurities and safety testing
- Vector genome titre, infectivity and RCV testing
- Characterisation: phenotype, function and genetic integrity
- Vector genome and capsid characterisation; insertion-site considerations
- Potency assurance: mechanism of action and quality risk assessment
- Process design, control and in-process measurements for potency
- Lot-release potency assays and matrix/orthogonal approaches
- Rapid clinical development challenges and lifecycle improvement
- Potency-assurance strategy exercise
- Release panel design and limited-sample prioritisation scenario
- Manufacturing changes: types and triggers
- Change criticality and regulatory reporting
- Analytical comparability
- Process-performance data and predefined acceptance criteria
- Nonclinical and clinical bridging
- Platform knowledge limitations in comparability
- Scale-up versus scale-out
- Autologous network expansion
- Technology-transfer packages and method transfer
- Technology-transfer gap assessment
- Process characterisation and process performance qualification
- Aseptic process simulation and shipping validation
- Hold-time, freeze-thaw studies and continued process verification
- Comparability protocol workshop
- Real-time and accelerated stability studies
- In-use and post-thaw stability
- Container-closure integrity and potency-indicating methods
- Small batch and limited sample strategies; shelf-life extension
- Cryogenic and ultra-low-temperature storage
- Dry shippers, temperature mapping and excursion management
- Courier qualification and chain of identity during transport
- Receipt and reconciliation at the clinical site
- Storage at site, thawing and preparation
- Administration windows and device compatibility
- Site administration readiness exercise
- Product returns and destruction
- IND CMC content
- IMPD quality section and CTD Module 3
- BLA and MAA quality content; facility and inspection information
- Meeting briefing packages and CMC questions/responses
- Information requests, deficiency management and commitments
- Agency question-response exercise
- Commercial readiness and post-approval changes
- Comparability protocols and platform/prior-knowledge strategy post-approval
- Inspection readiness: data integrity and batch records
- Deviation and CAPA, supplier oversight and training
- CTD Module 3 mapping exercise
- CMC readiness review
- 📘 Bonus: Cell and Gene Therapy CMC and Regulatory Strategy eBook (Free with purchase)
Course Benefits
Free eBook
Get our exclusive eBook with every purchase - a complete companion guide to the course, yours to keep forever
CPD Points
Gain Continuing Professional Development (CPD) Points, accredited by The Faculty of Pharmaceutical Medicine of the Royal College of Physicians of the United Kingdom. These can be used to count towards the distance learning element of any scheme that comes under the umbrella of The Academy of Medical Royal Colleges or any other scheme for which there is mutual recognition.
Certification
Receive a personal certificate to show your subject knowledge on course completion.
Affordable
You get excellent value through our cost-effective prices. We can also offer you group discounts on larger purchases.
Flexibility
The course saves you time through the convenience of online availability. This lets you complete the interactive course at your own comfort.
Keep Up to Date
You will stay up to date with evolving FDA and EMA cell and gene therapy expectations, including developing CMC-flexibility thinking for CGT BLA development, as our training courses are constantly monitored, reviewed and updated.
Learn from Industry Experts
The course content has been developed by CGT CMC and regulatory affairs specialists to ensure that learners can apply an integrated, phase-appropriate CMC strategy across development and commercial lifecycle stages.
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