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)

Course Description

CfPIE’s course on CMC regulatory compliance strategy for cell and gene therapy medicines navigates the complexities of Advanced Therapy Medicinal Products (ATMPs). This comprehensive, 20-hour program helps teams develop effective, risk-based strategies for unique living medicines.


This cell and gene therapy regulatory course applies chemistry, manufacturing and control (CMC), Good Manufacturing Practices (GMP) and Quality activities across clinical development to gene therapy CMC requirements. It also emphasizes the critical importance of communicating regulatory strategy with authorities at CMC-focused meetings.

Why This Course Matters

There has been a surge in the clinical development of Cell and Gene Therapy Products (CGTPs) in both the EU and the U.S. With the FDA projecting a significant increase in annual approvals for CGTPs, the market continues to expand rapidly despite complex regulatory hurdles and slow approval processes.


Key challenges facing the industry include:


  • Evolving ground rules: Unlike non-living biopharmaceuticals, the ATMP manufacturing compliance landscape for living medicines is still maturing.
  • Expedited pace: Clinical timelines place intense pressure on CMC teams to rapidly develop process controls.
  • Regulatory risk: Deficiencies in CMC regulatory compliance strategies have resulted in devastating clinical holds and market delays.



Patients in these clinical development programs need to be protected by common sense CMC, GMPs and Quality System principles. This course will thoroughly discuss and examine available guidance documents from the EMA and FDA for CGT medicines.

Who Should Attend

This course serves professionals seeking cell therapy quality systems training. It is essential for cell therapy companies and large biopharmaceutical companies that are developing and implementing advanced therapy regulatory strategies for commercialization.


Roles that benefit most from this training include:


  • Senior management and directors
  • Project planners and supervisors
  • Regulatory affairs personnel
  • Manufacturing and quality personnel
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Learning Objectives

  • 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 and 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 and 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 and 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


FAQs

  • What regulatory structure regulates gene therapy?

    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 and 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 and Gene Therapy manufacturing processes.


    Learn how to avoid major delays in clinical development or market approval due to an ineffective CMC regulatory compliance strategy.

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


    Overview of the Cell and 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  

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

    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.


  • What is the minimum score to pass the CMC Professional Exam?

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

Navigate the Future of Medicine With ATMP Compliance

Ready your regulatory strategy for the next generation of therapeutics. Add this course to your cart and explore specialized approaches to ATMPs. To request tailored training sessions, call our team at 610-648-7550 or contact us online today.

Certification Tracks

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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