Navigating ATMP Development - Online Training Recording
- From Bench to Bedside -
Course No. 23129
Speakers
Dr Sabine Hauck
dequra pharma consult hauck, Chair of ECA ATMP Interest Group
Dr Katja Aschermann
Astator
Dr Roland Pach
Roche
Prof Dr Sven Stegemann
DWI Leibniz-Institut für Interaktive Materialien e.V.
Dr Rainer Gnibl
GMP Inspector
- Current regulatory requirements (EMA, ICH, GMP) along the ATMP development process - Strategies for CMC, validation, QbD and scale-up - Understanding the roles of QP, development teams and clinical units - Practical case studies
Target Group
This training targets professionals in ATMP development, including scientists, QA/QC experts, regulatory specialists, and manufacturing leaders seeking practical GMP, CMC, and regulatory guidance from early phase to Scale-up.
Objectives
The aim of the training is to provide participants with fundamental, practical knowledge along the entire ATMP development process - from preclinical research to clinical application and GMP-compliant manufacturing. The content covers regulatory requirements (EMA, ICH, GMP), CMC strategies, process development, analytics, validation and quality assurance. Participants will learn how to overcome typical challenges, avoid typical mistakes and efficiently integrate regulatory and scientific requirements into everyday development work.
Background
The development of advanced therapy medicinal products (ATMPs), including gene therapies, cell therapies, and tissue-engineered products, is characterized by a high level of scientific, technical, and regulatory complexity. These innovative medicinal products must comply with stringent and ever-changing regulatory requirements, as set out in various European and international guidance documents. Examples include the EMA Guideline on “Quality, non-clinical and clinical aspects of gene therapy medicinal products” (EMA/CAT/80183/2014), The guideline on “Human cell-based medicinal Products” (EMEA/CHMP/410869/2006), EudraLex Volume 4 Part IV for ATMPs and cross-cutting ICH guidelines, such as ICH Q8–Q10 (quality by design and pharmaceutical quality systems) and ICH Q14 (analytical procedure development).
The transition from preclinical research to first-in-human trials and later market authorization – often referred to as the “bench to bedside” process – requires a development strategy that integrates regulatory foresight, robust CMC (Chemistry, Manufacturing and Control) planning, and comprehensive quality oversight from the earliest stages. Particular challenges in ATMP development include raw material qualification, platform-independent process validation, aseptic processing strategies, and the implementation of analytical approaches in accordance with ICH Q14 principles. Upscaling and tech transfer into GMP-compliant manufacturing environments require strategic foresight and alignment with GMP for ATMPs (EudraLex Vol. 4 Part IV).
This training course provides participants with a practice-oriented understanding of the end-to-end ATMP development process. It offers in-depth insights into regulatory expectations, process and analytical development, validation strategies, and the specific responsibilities of the Qualified Person (QP) under EU legislation. Participants will develop confidence in applying risk-based approaches (as outlined in ICH Q9) and addressing critical regulatory interfaces during development and scale-up. The course provides professionals with the strategies and regulatory expertise needed to successfully implement, ensure GMP compliance and ultimately authorize ATMPs in a dynamic and highly regulated environment.
Programme
From Phase 1 to Scale-up: GMP Requirements for ATMPs
Regulatory landscape: EMA guidance, ICH and GMP
Stability program
Challenges of IMPs and IMPD
Raw Material Selection in ATMP Development
Why it matters
Regulatory requirements
Selection criteria
CMC Strategies for ATMPs: Process Development and Quality Oversight
Quality oversight & regulatory
Addressing specific challenges in ATMP CMC
AAV case study
Qualification & Validation Challenges in ATMP Manufacturing Systems
Cleanroom qualification
Basics of process and cleaning Validation
Aseptic validation (media fill)
Validation life cycle
Analytical Development for ATMPs - Implementing ICH Q14 in Practice
Approaches for analytical procedure development
Enhanced approach in Theory
Practical examples
Applying QbD principles in ATMP development – Challenges and Opportunities
QbD (ICH Q8) in pharmaceutical product development to assure reproducibility by product and process understanding
Differences and communalities of small molecules, biologics and ATMP products
Potential of QbD to improve ATMP processing and control for point-of-care manufacturing
The QP’s Role in ATMP Development and Lot Release Under EU Law
GMP requirements for the QP role
Managing special cases specific to ATMPs
Communication between QP, manufacturing, and clinical teams
Towards “First-in-Human” – Considerations for early Clinical Trial Design of ATMP
Preclinical studies and data to support the appropriate design for the “first-in-human” clinical Trial
Traditional and evolving clinical study designs to proof safety and efficacy
Prerequisites and considerations to enter into a “first-in-human” trial
Scaling Up ATMP Manufacturing
Key aspects of scaling up ATMP
Examples & challenges of scaling up strategies
AAV case study
How to handle Deviations
What is a deviation and how to evaluate criticality
Core characteristics of an efficient deviation handling system
Root cause investigations
Change Control in agile ATMP Development Environments
Managing change control throughout the product LifeCycle
The change control process in an agile environment
Risk-based Approaches: How to do it right
Understanding the Risk-Based Approach in development of ATMPs
Risk assessment tools and the link to ICH Q9
Examples for implementation of risk-based approach
Bridging Development and GMP
Common pitfalls in ATMP tech transfer between R&D and GMP
Establishing robust documentation and traceability early on
Role of development reports, knowledge management, and comparability data
Further Information
Technical Details To participate in an on demand training course or webinar, you do not need any software. The recordings are made available via a streaming server. In general, the recording is provided in MP4 format, which any PC (Microsoft Windows, Apple IOS) or tablet can easily display.
Timing and Duration When you register for the on demand Training course or webinar you can decide at what date you want to follow the training course online. For a 1-day training course you will have 2 days in which the stream is available (for 2-day training course 3 days and for a 3-day training course 4 days). Within in this timeframe you can start & stop the stream according to your needs.
In time before the scheduled date (your desired date) you will receive an e-mail from us with a link for direct participation as well as your log-in data.
Please be aware: The recording does not include the Q & A sessions.
Training Course Documentation and Certificate The presentation will be made available as PDF-files via download during the online training course. After the successful completion of the online training, you are able to download the certificate of attendance.
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