Cell therapy CMC and quality control

Cell & Gene Therapy Insights 2021; 7(4), 571–573


Published: 13 May 2021
Christiane Niederlaender

Christiane Niederlaender. has spent 9 years at the UK medicines regulator MHRA, most recently as the Unit Manager of the Biologicals Unit. In that capacity she chaired and oversaw all formal scientific advice meetings and had in-depth involvement of Brexit preparedness. At the same time Christiane was a Senior Assessor and the UK delegate at the EMA committee for advanced therapies (CAT). She was UK CAT Rapporteur responsible for managing the portfolio of all UK ATMP products (cell and gene therapies). She was also the Rapporteur for the EMA CAT Gene Therapy Guideline and quality lead for gene therapy section of new draft guideline for investigational ATMPs. She was a drafting group member in all other quality related ATMP drafting groups, including GMP for ATMPs. Prior to joining MHRA, Christiane worked with the UK regulator for the European Tissues and Cells Directive as a quality manager and inspector. Christiane has experience in CMC and regulatory strategy for all types of biological products, with a particular focus on gene and cell therapies, novel biotherapeutics, biosimilars and vectored vaccines. While at MHRA, she was the principal quality/CMC assessor for 10 centralized MAAs, including two full dossier applications for ATMPs as sole UK quality assessor (including CAR-T, see publications). She has in depth knowledge of all European procedure types, both centralized and MR/DCP, and can give advice on ATMP classification, ODD and the PRIME program. As an ex-member of the EMA CAT committee, Christiane is familiar with the thinking of all European regulators in the ATMP space and is used to dialogue and negotiation with many regulatory agencies, as well as presenting her thinking during committee discussions. Christiane undertook her undergraduate studies in biochemistry, neurobiology and molecular biology at Heidelberg University in Germany before obtaining her PhD in molecular developmental neurobiology at Kings College London in the UK. Christiane has published her academic research in many high impact journals, including Nature.