Mesenchymal stem cells (MSCs) are attractive candidates for therapeutic applications, especially in the field of regenerative medicine [1] because – in contrast to embryonic stem cells – they do not pose ethical issues, they can be isolated from various tissue sources, and they reduce the risk of rejection reactions. The doses of human MSCs (hMSCs) needed for clinical trials are estimated at between one and 200 million cells per patient, depending on the disease being tackled [2]. One of the most important challenges in providing hMSCs for curative use is the production of large quantities of cells in a robust manner.

The case study of BioMarin Fast-Track project with Verdot Ips²

This paper reports a very fruitful collaboration between a Biopharmaceutical company, BioMarin International Ltd and a manufacturer of purification equipment, VERDOT Ips², which contributed to a fast start-up of an installation for manufacturing a recombinant human tripeptidyl peptidase 1 (rhTPP1), for the treatment of neuronal ceroid lipofuscinosis Type 2 (CLN2) disease, also known as Batten Disease.

Many industries including those associated with generation of power and the manufacture of glass, steel and petrochemicals etc. have demonstrated that continuous manufacturing provides significant benefits and advantages, ranging from reduced capital and operating expenses to greater efficiency and product quality and consistency.

Following to questions raised by French Biotech industry on GMP part I and II cleavage proposed by inspectors regarding biological production process, the objective of this article is to propose a clarification in the particular case of biological active ingredients when formulation is performed during Drug Substance manufacturing instead of during Drug Product manufacturing.
This apply fully to recombinant proteins and monoclonal antibodies and case by case to other biotechnology manufacturing processes and innovative therapeutic (vectors of gene therapy and vaccines,…)