GMP Manufacturing & Solutions
GMP, or Good Manufacturing Practices, are a set of regulations applicable to the manufacturing of health product candidates, especially medicines intended for human use, such as UCART product candidates. A company is required to comply with GMP regulations in order to be granted a license from governmental regulatory agencies to manufacture pharmaceutical product candidates. The Manufacturing department takes manufacturing processes established at the R&D level, converts them to GMP, and ensures their deployment with GMP compliant raw materials and environments. The department is responsible for the manufacturing of clinical trial material (“CTM”), making it available for clinical studies and afterwards, and also for the manufacturing of final GMP commercial cellular gene therapy products. The team interacts internally with different departments ranging from development and planning, to regulatory and legal, as well as externally with raw materials contractors or GMP manufacturing contract organizations.
Our proprietary manufacturing process
Through our manufacturing process, therapeutic UCART product candidates are made from healthy, tested and qualified donor T-cells; unlike autologous CART approaches all derived from patient samples. This “off-the-shelf” approach leads to lower production costs. In addition, our process – powered by TALEN® and our proprietary PulseAgile electroporation technologies – inactivates genes in a highly efficient manner that avoids harming T-cells during processing. As a result, we can manufacture quality UCART products with high yields --and potentially in bulk. We expect that T-cells from one healthy donor, and one manufacturing run of UCART, could be used to create hundreds of doses of product and more when scaling up the process. These efficiencies may not only reduce costs to patients but also lead to competitive gross profit margins.
CELLforCURE is producing clinical batches of UCART123, our first wholly owned UCART product candidate, to meet the needs of the Phase 1 clinical trial, as part of the development of UCART123 in malignancies, such as acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN).