Gene therapies are «once in a lifetime» treatments that use vectors to genetically modify cells of interest and confer them with a function they did not possess before. They can address broad sicknesses such as HIV or several forms of cancer but also orphan diseases and will completely disrupt the way therapeutic means are envisioned. The gene therapies we focus on are applied by extracting the cells to modify them outside of the body.
To apply ex vivo gene therapy a patient’s white blood cells are extracted by apheresis in hospitals and cryogenized. The sample is packaged and sent to a manufacturing facility. Cells of interest, generally hematopoietic stem cells or lymphocyte-T are isolated from the rest of the biological fluid. They are then modified by a lentiviral vector to alter their genetic code in order to give them a function of interest. Finally they are cultivated to expand their number before being concentrated, frozen and sent back to the clinic to be reinjected in the patient.
The manufacturing procedure, for a single patient, is performed in a clean room environment by a team of technicians and requires several days of work. One of the difficulties resides in complex standard operating procedures and the inherent imperfection of human operators that negatively impacts the quality and reproducibility of the final therapeutic product. Each step of the process is made in a different instrument which implies that transport of the product from one device to another increases the chance of contamination and sample degradation. Furthermore, the high quality clean rooms are expensive and tedious to maintain. Finally, transport of the final product is a source of risk as it can be lost, frozen in an unsatisfactory fashion.
We propose to automatize the manufacturing of ex vivo gene therapies by providing an instrument that fabricates, at the patient bedside from his white blood cells, the final therapeutic product. With such an instrument it is possible to reduce treatment duration by two days, remove transportation, decrease the number of operators by nine and increase reproducibility of the process. High-grade clean rooms are no longer necessary in standardized applications. Based on a novel processing chamber, able to incorporate different magnetic cell selection means as well as several types of vector based procedures, we proposes a versatile platform that can manufacture any type of gene therapy for a large selection of cells. Technologies using vectors to apply hematopoietic cell transformations, CAR T-cell and CRISPR/Cas9 can be used in the system.
The technology being in the process of patenting no details can be currently given.