ATMP - Manufacturing, Quality and Safety

Objective

This conference track is aimed at all those who develop and manufacture cells, tissues, cell- and tissue-based products and ATMPs. The conference will address manufacturing challenges, e.g. GMP regulations, but also quality control issues, appropriate ways to maintain, assure and control the expected quality. Experienced speakers from the field of ATMP will explain the current
requirements and report on their experiences during inspections and the implementation in the company.

Target Audience

This conference is aimed at all persons who

  • are involved in the extraction and manufacture of Cells, Tissues and ATMPs
  • are responsible in quality assurance and control of Cells, Tissues and ATMPs
  • are responsible for microbiological or analytical testing
  • perform inspections or audits of ATMPs facilities
  • deal with the authorisation

 

Background

Modern systems of regenerative medicines, such as cells and tissues or ATMPs (gene therapeutics, somatic cell-based products and tissue-based products) represent an innovative group of drugs that is becoming increasingly important. With the entry into force of several regulatory guidelines e.g. of the European Directive EC 1394/2007 for ATMPs, such products were classified as medicinal products and must therefore comply as such with the EU requirements for medicinal products. Although the biopharmaceutical industry has considerably intensified its activities in this field, 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 regulaquality, safety and GMP aspects and approval. This is also forced by frequently given manufacturing conditions, e.g. the open manipulation of cells and tissues, which are necessary for obtaining such products on a medical/surgical level or by the short shelf life of
the obtained final product. Challenges for small batch manufacturing, rapid testing and analysis and storage are only some of the challenges for such short shelf life products in terms of:

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

Detailed Programme

Presentations - 28 March 2023

Vectors for ATMPs – Stabilization by Formulation Development
Dr Sabine Hauck, Leukocare

  • Algorithm based formulation development and in silico long-term prediction
  • Stabilization of viral vectors beyond the expected
  • The next generation: LNPs as vectors

ATMP: Market, Manufacturing and Challenges
Dr Mohamad Toutounji, Molgenium

  • Global market overview of ATMP
  • ATMP Clinical trials
  • Advanced manufacturing processes and scalability
  • Development of new methods for quality control of ATMP
  • Regulatory pathways and challenges

GMP for ATMP
Dr Rainer Gnibl, Government of Upper Bavaria

From Process Transfer to clinical / commercial Manufacturing of Cell and Gene Therapy Products – Experiences of a CDMO
Kati Kebbel, Fraunhofer Institute for Cell Therapy und Immunology

  • Execution of Process transfer / process setup from CDMO point of view
  • Interaction in a pharmaceutical system
  • Process documentation and Manufacturing license
  • Exemplary challenges of a CDMO like raw materials, ramp up, phase out

Cell based ATMPs: A success story
Dr Katja Aschermann, TETEC

  • Tissue engineered products
  • Regulatory Landscape
  • Challenges of autogenous cell-based ATMPs
  • Case study: Novocart® Inject
Presentations - 29 March 2023

Individual stringent Mini Isolators for autologous Cell Therapy
Didier Meyer, DM

  • Common Strategy to avoid Cross Contamination
  • A decentralised scale-out concept to be implemented in hospitals
  • How a dual temperature Class C or D decentralised platform of a maximum of 10 mini-isolators from donor/patient leukapheresis allows within one to three weeks to take care of more than 100 patients a year using CAR- T techniques and making it affordable

Case study: QC Test Profile and Release Strategies for ATMP
Patrick Büschor, Cutiss
Stephane Gumy, PMSystems

  • The QC profile of ATMPs
  • The challenges of releasing extemporaneous ATMPs
  • Different release strategies for ATMPs (extemporaneous finished products, reconstitued vaccine, ...
  • The use of potential contaminated ATMPs or OOS products in the frame of clinical trials

Exceptional Provision of AT(I)MPs affected by OOS Results
Dr Rüdiger Alt, Novartis

  • Introduction & regulatory framework
  • Process and workflow for exceptional provisions
  • Health Authority interactions
  • Opportunities and limitations of chapter 11.5

Freezing and Thawing
NN

Establishment of Quality Management System (QMS) for Cell Therapies
Erik Steffensen, Novo Nordisk

  • Cell therapies - When living human cells are the pharmaceutical product
  • Similarities and differences between conventional medicines and cell therapies from a scientific and regulatory point of view
  • To what extent considerations from the development and manufacture of conventional medicines and biologics can be used in the design of the QMS for cell therapies
  • Design principles and the implementation of a QMS for cell therapies

The Assessment of Process Equipment Related Leachables in Cell and Gene Therapies
Dr Armin Hauck, Sartorius Stedim Biotech

  • The specialties with extractables and process equipment related leachables (PERLs) in cell and gene therapy
  • Exposure estimations for therapeutic cells and patients established with PERL-model calculations and digital twins
  • The use of a high throughput cell painting assay to screen for toxic and/or detrimental effects of PERLs on therapeutic cells
x