Conference: Cell and Gene Therapies/ ATMPs - Quality and Safety

Objectives

This meeting is aimed at manufacturers and developers of cells, tissues, cell- and tissue-based products or ATMPs and deals with microbiological and analytical quality requirements, suitable methods and test systems and their implementation and validation. Representatives from authorities and colleagues from small-scale and industrial manufacturing and academic institutions will explain the current regulatory requirements and report on their experiences during inspections and implementation in the company.

Background

Modern regenerative medicine systems such as cells and tissues or ATMPs (gene therapeutics, somatic cell-based products and tissue-based products) represent an innovative group of medicinal products that is becoming increasingly important. With the entry into force of several regulatory directives, e.g. the European Directive EC 1394/2007 for ATMPs, such products have been classified as medicinal products and as such must comply with EU requirements for medicinal products. Although the biopharmaceutical industry has significantly intensified its activities in this area, many of these products are developed and manufactured at universities, hospitals and in small and medium-sized enterprises. These university or medical roots lead to special challenges for the respective institutions as well as for the regulatory authorities in meeting compliance requirements for quality, safety and GMP aspects and approval. The frequently given manufacturing conditions also contribute to this, e.g. the open manipulation of cells and tissues necessary for obtaining such products on a medical-surgical level, or the short shelf life of the obtained end product. And potentially there are always conflicts when it comes to the relevance of different guidelines, e.g. when an Annex 1, or an Annex 2 or a WHO Guideline does not harmonise with the ATMP Guideline.

But also rapid tests and analyses are a challenge for such products with a short shelf life in terms of

• Comparability with Compendial Methods
• Sensitivity and Robustness
• Suitability Testing and Validation
• Variability

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Conference Registration

Registration

Target Audience

This conference is of interest to professionals from Biotechnological & Biopharmaceutical Companies, Contract Service Laboratories, Academic Research Institutions and Organizations, Government Agencies, Cell Culture Collections Supplier Detection Systems with responsibilities in

• Manufacturing
• Quality Assurance
• Quality Control
• Regulatory Affairs
• Research & Development
• Process Development
• Validation

Moderation

Dr Sabine Hauck, Chair of the ECA ATMP Board
Dr Ulrike Herbrand, Charles River Laboratories

Tuesday, 26 November 2024

Key Note on 26 November: The Promise and Challenges of In Vitro and In Silico Models in Drug Development
Dr Julia Schüler, Charles River Laboratories
The presentation will highlight important developments in the drug development technology landscape influenced by the concept of 3R and the evolving legal landscape. General characteristics of the different applications, their translational relevance as well as adoption drivers will be discussed. Case studies from oncology drug development will help to elucidate these trends and their impact on future processes.

Implementation of ICH Q14 and USP <1220>: A challenge in the Highly Competitive mRNA Vaccine Field
Dr Isabelle Moineau, AKTEHOM & Dr Marc Francois-Heude, Sanofi

• Implementation of an optimized Life Cycle Management process for analytical methods
• Case study: design of analytical methods to Ongoing Procedure Performance Verification

Spilling the Tea on a Robust CCS for ATMPs
Marsha Steed, Steed MicroBio

• Establishing a CCS for ATMPs
• Are there regulations for CCS for ATMPs?
• Material transfer contamination controls for ATMPs
• Use of isolators for ATMP manufacturing
• Particulates in ATMP products and impact of single use systems

European Pharmacopeia Perspective
Dr Solène Le Maux, EDQM

• Activities of the Ph. Eur. in the ATMP field
• Focus on the new approach to gene therapy texts
• Review of the cell-based preparations chapter under elaboration
• Updates on alternative rapid microbiological methods

What is the Value of Design-of-Experiment Approaches in the Development of Cell-based Potency Assays?
Dr Johannes Solzin, Boehringer Ingelheim

• Comparison of different optimization strategies and different DoE-approaches for bioassays within development of biopharmaceuticals
• How to minimize number of experiments and maximize statistical power of a DoE
• Choosing the right response for DoE: Which parameter(s) describe the performance of a bioassay?
• Case studies of assay development: from potency assays for antibodies to new infectious titer assays for virusesbased ATMPs

Key Insights about CAR-T Therapy from Concept to Clinic
Dr Daniela Rozkova, SCTbio

• From tech transfer to clinical production
• Navigating challenges & innovations in Autologous CAR-T Cell Therapy
• Lessons learned

Critical Quality Attributes of AAV based GT Products
Dr Roland Pach, Roche

• Industrial perspective on the determination of CQAs
• Impact on techniques and strategies
• Product related impurities and potency