Division Medical Biotechnology
The division oversees advanced therapy medicinal products (ATMPs) such as gene therapies, somatic cell therapeutics, tissue-engineered products (TEPs), but also oncolytic viruses and human tissue preparations, from the approval of clinical trials through to the marketing authorisation or approval of the medicinal product. One additional area of focus is offering scientific and regulatory advice to companies, research groups, tissue establishments, state authorities and the world of politics – because ATMP often demand customised solutions. Experts from the division are represented on the relevant committees and working parties of the European Medicines Agency (EMA), the European Directorate for the Quality of Medicines (EDQM) and the World Health Organization (WHO), the European Society of Gene and Cell Therapy (ESGCT), the German Society of Gene Therapy (DGGT) and the German Stem Cell Network (GSCN), and help shape the regulation of ATMPs. They draw up and comment on regulatory guidelines for the development and testing of ATMPs. The division’s research work focuses on fundamental concepts in the development and testing of ATMPs that are relevant to their efficacy and safety.
Head of Division’s Research Group
The group carries out research into the basic biological and biotechnological principles underlying the development of ATMPs. Adverse reactions, which can hinder the successful use of ATMPs in clinical practice, represent a particular challenge for developers and regulators. For example, therapeutic gene transfer vectors may, during the integration of genetic material in somatic cells, deactivate a cellular tumour suppressor gene or activate a proto-oncogene. To reduce this risk, the research group is developing experimental therapeutic approaches based on transposons – jumping genes – for targeted gene transfer to safer locations in the genome, so-called “safe harbours”. In addition, they are exploring targeted methods of modifying the genome of somatic cells or repairing defective genes using designer nucleases such as CRISPR/Cas. The researchers evaluate these approaches in therapeutically relevant cell types such as human T cells and in appropriate animal models. The goal is the successful translation of gene therapies into clinical practice.
Advanced Therapy Medicinal Products, Tissue Preparations Section
The section coordinates the division’s regulatory work and in particular assesses all clinical aspects, but also the quality and non-clinical testing of ATMPs and tissue preparations. One of the section’s experts is Chair of the Committee for Advanced Therapies (CAT) at the EMA, which in addition to the actual committee work also entails many related activities. The section’s duties also include handling major regulatory issues, supporting management and providing policy advice.
Non-viral Gene Transfer Medicinal Products Section
The section assesses documents on the quality, non-clinical testing and Environmental Risk Assessment (ERA) of applications for the approval of clinical trials of gene therapies. The section advises applicants on these aspects in EMA Scientific Advices and scientific consultations by the Paul-Ehrlich-Institut (PEI) and provides support for centralised marketing authorisation procedures of gene therapy medicinal products. Its experts take part in GMP inspections of the relevant state authorities and represent the PEI on committees and working parties of the EMA and EDQM. The section’s scientists carry out testing-related research into the safety and efficiency of gene transfer systems.
Tissue Engineering, Somatic Cell Therapeutics Section
The section assesses applications for the approval of clinical trials and centralised European marketing authorisation procedures for human somatic cell therapeutics and tissue engineering medicinal products. The section provides advice on issues relating to the quality and non-clinical testing of these medicinal products in PEI scientific consultations, and its experts take part in GMP inspections of state authorities. It represents the PEI on the CAT and in expert groups of the Central Authority of the Länder (ZLG) and WHO.
The scientists carry out research into the safety and efficacy of cell-based medicinal products – especially with regard to the differentiation of human stem cells. The research focuses on the influence of endogenous human transposable elements on the integrity of the genome and on physiological processes in human stem cells – starting from cell types obtained by differentiation.
Non-vital Tissue Preparations, Xenogeneic Cell Therapeutics Section
The section assesses applications for the approval and marketing authorisation of human tissue preparations and xenogenic medicinal products. It provides scientific advice to applicants and pharmaceutical companies. Its experts take part in GMP/GFP inspections of state authorities and represent the PEI in national expert groups of the state authorities and in international expert groups of the EU, EMA, EDQM and WHO. Additionally the section records and evaluates the data in accordance with the German Transplantation Act (TPG) and the Preimplantation Diagnostic Regulation (PIDV).
The scientists carry out research in the field of infection risks from xenotransplantation – the transplantation of animal organs as well as animal tissue and cells into humans – by characterising the microbiological and virological risks. The focus is on an analysis of the pathomechanisms of porcine endogenous retroviruses (PERVs) as well as other porcine pathogens of genetically modified donor pigs. The researchers are developing detection and control methods and defining test criteria to enable the use of tissue, cells and organs from pigs for xenotransplantation into humans.
Viral Gene Transfer Medicinal Products Section
The section carries out research into CAR T cell therapy. The focus is on investigations into viral gene transfer, but also on the establishment and standardisation of animal models for predicting CAR T cell-associated adverse reactions and understanding the underlying pathomechanisms. The staff supports the division’s regulatory duties in the area of gene therapy, for example by taking part in consultations, presentations and the evaluation of applications for clinical trials.