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