Industries: Pharma / Biotech

CMC Regulatory Compliance Strategy for Cell & Gene Therapy Medicines

Course Director: John Geigert, Ph.D.

Course Fee: $2150.00 Regular Registration / $1950.00 Early Bird (30 Days in Advance)

  Accreditation: ***RAPS Approved Course***

PharmaTrain Recognized Course    


Course Description -

This course will help those involved with cell and gene therapy medicines (also referred to as ATMPs, Advance Therapy Medicinal Products) 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 these manufacturing processes and products. The critical importance of communicating CMC regulatory compliance strategy with the regulatory authorities at CMC-focused meetings will be stressed.

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 companies, and now even large biopharmaceutical companies, are planning or have already entered into manufacturing these genetically engineered viruses and/or human cell products for clinical studies. While off to a slow start of market approval, the FDA has projected that by 2025 there might be up to 10-12 new Cell and/or Gene Therapy products approved for the market each year.

While the CMC strategy, ground rules for Good Manufacturing Practices (GMPs) and Quality Systems are well established for the nonliving protein biopharmaceuticals, the CMC strategy and GMP manufacturing process ground rules for the more complex, living CTG 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.

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.

Who Should Attend

This two-day CMC regulatory compliance course is designed for senior management, directors, managers, supervisors, project planners and professional staff seeking to develop or implement a Chemistry, Manufacturing & Controls (CMC) regulatory compliant strategy for Cell & Gene Therapy products. Typical attendees include: Senior Management, Project Managers, Regulatory Affairs, Manufacturing, and Quality and Development personnel.

Course Agenda

Day One

Overview of the Cell & Gene Therapy Landscape

  • Increasing diversity of Cell & Gene Therapy medicinal products
  • Defining critical terminology: CGTP, ATMP, CAT, OTAT, RMAT, …
  • The regulatory authorities (FDA, EMA) involved and the regulatory review pathways (IND to BLA; IMPD to MAA)
  • Major CMC challenges working with living CGT medicines

CGTP CMC Regulatory Guidance

  • Critical necessity of a risk-based CMC approach during clinical development
  • Key design elements leading to an effective CMC regulatory compliant strategy
  • Effectively managing the ‘minimal CMC regulatory compliance continuum
  • FDA/EMA CMC guidances for Cell & Gene Therapy products

Day Two

Applied Risk-Managed CMC Regulatory Compliance Strategy

  • Detailed roadmap for manufacture of a genetically engineered viral vector – from source material to final drug product
  • General roadmap for manufacture of a cell-based medicine – from source material to final drug product
  • CMC experience/knowledge carryover from the biological industry applied to CGT medicines – what is value added, what are the minefields
  • Challenge of confirming product comparability after a process change

Critical CMC-Focused Strategic Interactions with Regulatory Authorities

  • Maximizing the value of CMC discussions/meetings with the regulatory authorities for CGT medicines under clinical development
  • Avoiding critical delays in clinical development and market approval due to an ineffective CMC regulatory compliance strategy
  • Opportunities for CMC-focused meetings for CGT medicines with FDA and EMA
  • Regulatory submission content for CGT medicines

Learning Objectives

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

  • Gain a solid understanding of the CMC regulatory compliance requirements and expectations (FDA, EMA, ICH) for Cell & Gene Therapy products 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


RAPS | Regulatory Affairs Professionals Society CfPIE is a Regulatory Affairs Professional Society (RAPS) RA Professional Development provider.

CfPIE is committed to enhancing the ongoing professional development of regulatory affairs professionals and others through appropriate regulatory affairs learning activities and programs.  CfPIE has agreed to follow RAPS-established operational and educational criteria.  Click here for the list of courses reviewed and approved by RAPS.