GMP Manufacturing & Solutions
GMP, or Good Manufacturing Practices, are a set of regulations applicable to the manufacturing of health products, especially medicines intended for human use, such as UCART products for example. A company is required to comply with GMP regulations, in order to be granted from governmental regulatory agencies, its license to manufacture pharmaceutical products. The GMP Manufacturing & Solutions department takes manufacturing processes established at 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, planning, to regulatory and legal, as well as externally with raw materials contractors or GMP manufacturing contract organizations.
Our manufacturing process
Through our manufacturing process, we obtain therapeutic UCART product candidates from healthy, tested and qualified donor T-cells, rather than 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. This could enable us to manufacture in bulk, and 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. In 2015, the manufacturing process of our first UCART product candidate, UCART19, was further developed, pursuant to GMP quality standards, and was deployed in a GMP environment. Production was started to meet the needs of the first phase clinical trials, as part of the clinical development of UCART19 in adult and pediatric acute lymphoblastic leukemia as well as in adult chronic lymphocytic leukemia. For that production campaign, we relied on external GMP capacity at CELLforCURE, a contract manufacturing organization. On October 28, 2015, we announced that we completed a series of three production runs of UCART19.
On January 19, 2016, we announced that, pursuant to a second agreement, CELLforCURE will also produce clinical batches of UCART123, our first wholly owned UCART product candidate, to meet the needs of the first phase clinical trial, as part of the development of UCART123 in malignancies, such as acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN). On January 19, 2016, we announced that, pursuant to a second agreement, CELLforCURE will also produce clinical batches of UCART123, our first wholly owned UCART product candidate, to meet the needs of the first phase clinical trial, as part of the development of UCART123 in malignancies, such as acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN).