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

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

Programme

Key Note on 21 November: The Impact on the Revised Annex 1 for Rapid Microbiological Methods Implementation
Dr Michael Miller

Key Note on 22 November: Preparedness in pandemic vaccine manufacturing and deployment
Prof Dr Isabelle Bekeredjian-Ding

Analytical Challenges in Development of Cell and Gene Therapies
Alicja Fiedorowicz, Dark Horse Consulting

Challenges of a Point-of-care Model for Cell Therapy from an Analytical Perspective
Matthias Heemskerk, CellPoint, a Galapagos company

Potency Testing for ATMPs
Dr Sascha Karassek, Charles River Laboratories

Analytical Methods to support mRNA-LNP Formulation Development
Dr Sabine Hauck, Leukocare

Achieve Lower LLOQs for siRNA Quantification in Plasma using Microflow LC
Dr Ferran Sanchez, Sciex

Low Energy Electron Irradiation (LEEI)
Dr Sebastian Ulbert, Fraunhofer-Institut für Zelltherapie und Immunologie

MucoCell® – a New ATMP for a high-medical need Urological Indication
Dr Markus Fido, MFI Consulting

Selection of Appropriate Methods for Detection of Microbiological Contaminations in ATMPs
Dr Stefanie Bayer, Labor LS

Innovations in Quality Control for Cell and Gene Therapy using Digital PCR
Nico Chow, Thermo Fisher Scientific

Development of a Digital PCR-based system for the Detection of Residual DNA in Pharmaceutical Products
Dr Nicole Paland, Minerva

Use of Bact/Alert 3D Instrument for Release Testing of Cell and Gene Therapy Product
Nicolas Weber, Novartis

Mycoplasma Testing & Evaluation for ATMPs – lessons learned!
Olga Müller, Tetec

Challenges of Endotoxin Detection During Development of a Novel product
Dr Annika Böhm, Microcoat

Automating the Future of Cell and Gene Therapy: Streamlining Endotoxin Detection, Data Integrity and Compliance Solutions with recombinant Factor C
Dr Christian Faderl, bioMerieux