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CMC Regulatory Compliance Strategy for Cell & Gene Therapy Medicines

Learn all aspects of how to create a CMC-compliant program when it comes to the cell and gene therapy manufacturing process. This course provides a comprehensive overview of cell therapy and gene manufacturing, what cell therapy companies should expect when it comes to CMC regulatory compliance, and more.

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    "Great package of notes/slides provided. Good balance of slides, interactive discussions and breaks. Relevant, targeted content with lots of real cell and gene therapy examples included. "


    Kathryn R., Manager, Regulatory Sciences, Oxford Biomedica

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Course Director

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Course Fee

$2650.00 Regular Registration

$2450.00 Early Bird Pricing (Register 30 Days in Advance)

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Course Description

This 20 hour course is designed for life sciences professionals involved with the cell and gene therapy manufacturing process for medicines (also referred to as ATMPs, Advance Therapy Medicinal Products) looking to develop an effective risk-based Chemistry, Manufacturing & Control (CMC) regulatory compliance strategy for these unique living medicines. 


The full spectrum of CMC, GMP and Quality activities across the pathway of clinical development will be applied to the unique requirements of cell and gene therapy manufacturing processes and products. The critical importance of communicating CMC regulatory compliance strategy with the regulatory authorities at CMC-focused meetings will be stressed within the context of cell therapy companies and their regulatory requirements.


Why This Course Matters

There has been a surge in clinical development of Cell & Gene Therapy Products (CGTPs, Advanced Therapy Medicinal Products), both in the EU and the USA. 


Many start-up cell therapy companies, and now even large biopharmaceutical companies, are planning or have already entered into cell and gene therapy manufacturing to create genetically-engineered viruses and/or human cell products for clinical studies. While the FDA projects by 2025 there might be up to 10-12 new Cell and/or Gene Therapy products approved for the market each year — this segment of the market continues to experience a slow approval process.


Despite the well established nature of CMC strategies, Good Manufacturing Practices (GMPs), and Quality Systems for the nonliving protein biopharmaceuticals — the CMC strategy and GMP manufacturing process ground rules for the more complex, living cell and gene therapy products are still evolving. In addition, the expedited pace of CGT clinical studies places intense pressure on the CMC teams to rapidly develop and enhance the manufacturing process control systems for potential commercialization. Deficiencies in the CMC regulatory compliance strategy have resulted in recent devastating clinical holds and market approval delays for cell and gene therapy manufacturing companies.


Patients in these clinical development programs need to be protected by common sense CMC, GMPs, and Quality System principles. The available EMA/FDA regulatory authority guidance documents for the CGT medicines will be thoroughly examined throughout this course.

Who Should Attend

This 20 hour CMC regulatory compliance course is designed for senior management, directors, managers, supervisors, project planners and professional staff working at cell and gene therapy manufacturing companies seeking to develop or implement a Chemistry, Manufacturing & Controls (CMC) regulatory compliant strategy for the cell and gene therapy manufacturing process and products.

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  • Learning Objectives

    At the end of the three-day course attendees will:

    • Gain a solid understanding of the CMC regulatory compliance requirements and expectations (FDA, EMA, ICH) for Cell & Gene Therapy medicines during clinical development and market approval
    • Have the tools and understanding to develop and carry out an effective CMC regulatory compliance strategy for the company’s CGT product(s)
    • Understand the strengths and limitations of Quality by Design (QbD) and Quality Risk Management (QRM) for Cell & Gene Therapy manufacturing processes
    • Learn how to avoid major delays in clinical development or market approval due to an ineffective CMC regulatory compliance strategy 

  • Agenda


    Overview of the Cell & Gene Therapy Landscape

    • Discussion of the increasing diversity of the advanced therapy medicinal products
    • Defining critical terminology: CGTP, ATMP, CAT, OTAT, substantial manipulation, not for homogeneous use, …
    • Introduction to the regulatory authority systems (FDA, EMA) in place to regulate the evolving in vivo and ex vivo gene therapy and cellular therapy manufacturing processes and products: IND → BLA; IMPD → MAA

    Risk-Managed CGTP CMC Regulatory Compliance Strategy 

    • Current status of CMC guidance from regulatory authorities (FDA/EMA/PICS)
    • Critical necessity of a risk-based CMC approach during clinical development
    • Lessons learned from the existing biologic industry – what works well (e.g., aseptic processing, quality unit responsibilities, etc.) and what does not work well (e.g., personalized medicine vs inventory build, time pressures on QC/QA, etc.)
    • Quality by Design (QbD)/Quality Risk Management (QRM) approach to effectively manage the ‘minimum CMC regulatory compliance continuum’ 

    Applied Risk-Managed CMC Regulatory Compliance Strategy

    • Applied CMC regulatory compliance strategy for in vivo and ex vivo gene therapy and cellular therapy manufacturing processes and products
    • Raw Materials → Starting Materials → Production → Purification → Formulation → Drug Product → Administered Drug Product
    • Five (5) challenging CMC regulatory compliance issues for advanced therapy medicines
    • 1) Need for increasing ‘adequate and appropriate control’ (level of GMP control and maturity of the Quality System) of the manufacturing process across the advancing clinical development continuum 
    • 2) More than the drug product – ensuring the drug product is accurately and consistently administered to the patient 
    • 3) Importance of identifying product Critical Quality Attributes (CQAs), and then linking them to the identification of the manufacturing process Critical Process Parameters (CPPs) – sooner than later in clinical development – to establish an effective Control Strategy
    • 4) Challenge for CGTPs of confirming product comparability after manufacturing process changes – utilizing the three (3) risk-based considerations (nature of change, stage of clinical development, and stepwise approach)

    Applied Risk-Managed CMC Regulatory Compliance Strategy   (cont. from Second Day)

    • Five (5) challenging CMC regulatory compliance issues for advanced therapy medicines
    • 5) The critical impact that ‘Clinical Development Expediting’ for CGTPs has on the CMC regulatory compliance strategy

    Critical CMC-Focused Strategic Interactions with Regulatory Authorities

    • CMC, especially for advanced therapy medicines, can severely delay either advancing in clinical development or getting into the market – steps to avoid this
    • Critical importance in obtaining the needed regulatory authority’s input into the CMC regulatory compliance strategy
    • Maximizing the value of CMC strategy discussions/meetings with the regulatory authorities (FDA/EMA) for CGT medicines along the clinical development pathway  
    • Maximizing the value of CMC regulatory compliance discussions/meetings with the regulatory authorities (FDA/EMA) for CGT medicines during the BLA/MAA review and into market approval  

  • Testimonials

    "This was the most thorough course I have taken on this or related topics and the instructor was a great speaker and the material well organized.  The breadth of material covered and the invaluable course book provided as a reference. The instructor was also a great speaker, knowledgeable, and very engaging." 

    Nicole B., Associate Director Regulatory Affairs, Kriya Therapeutics


    “Course was well structured and content was very well organized.  I got an overview of the whole CMC process rather than just the part I am closely involved in. Section on Risk Managed Compliance was very interesting.

    Moira E., Senior Group Leader, PPD Ireland


    "Great package of notes/slides provided. Good balance of slides, interactive discussions and breaks. Relevant, targeted content with lots of real cell and gene therapy examples included."

    Kathryn R., Manager, Regulatory Sciences, Oxford Biomedica


    "I really enjoyed receiving examples I can take back to my company and use to forge a more compliant future. I think learning from others is the space is an underutilized tool."

    Julianna P., Manager, Research Quality Assurance, Astellas Pharma


Registrant Information:

Each person attending a course will be asked to set up an Attendee Profile Account during the registration process. Creating an Account helps you view your order history and manage your training programs. If you are registering for others, please set up an Account in the Attendee’s name. If you are registering more than one person, you’ll need to set up a separate account for each Attendee.

Frequently Asked Questions


Q: What regulatory structure regulates gene therapy?

A: Cell and gene therapy companies are governed by several regulatory agencies, including the FDA and CBER in the United States and the EMA and CAT in the European Union.


Q: How many years of experience in cell and gene therapy manufacturing do I need to be eligible to take the course?

A: No experience is required to take this course. Participants will learn the foundational knowledge required to better understand the relationship between CMC regulations and cell and gene therapy manufacturing.


Q: Is the Regulatory Compliance Course necessary to pass the CMC Professional Exam?

A: While this is a specific course in the certification track, it is not 100% required to earn the certification. Participants can use any combination of four courses in that track.


Q: What is the minimum score to pass the CMC Professional Exam?

A: Participants must achieve at least a 70% score to pass the CMC professional Exam.


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