Operational Milestone #2
We are developing a new stem cell transplant product, AB-110, to treat life-threatening, high risk blood cancers, such as lymphoma and leukemia. AB-110 is composed of human umbilical cord blood derived hematopoietic stem and progenitor cells co cultured and expanded with Angiocrine’s proprietary E-CEL UVEC cells. The early studies demonstrating the feasibility of this approach in animal models used product derived from small scale cell co-culture in tissue culture flasks. To manufacture the product at a scale suitable for treating patients, we devised a process that uses a closed system bioreactor that can rapidly produce sufficient material with minimal human intervention. This process is both economical and reliable. Our objective in the past quarter was to optimize this manufacturing process and to demonstrate, both in cell culture assays and in animal models, that the product generated in the bioreactor is comparable to the product generated in tissue culture flasks. We have now achieved a process to reliably manufacture the E-CEL UVEC cells in the closed, automated mini-bioreactor system, that will be utilized for clinical and likely commercial production. In addition, we systematically explored the many variables in the co-culture process and ultimately succeeded in demonstrating that we could reproducibly expand umbilical cord stem and progenitor cells over hundred fold, to numbers similar to what is expected of cell numbers from adult sources, and within two weeks or less, which is regarded by transplant physicians as a time frame that would be compatible with clinical practice. We then succeeded in demonstrating that cells derived from the two processes are indistinguishable in their cellular composition, their content of stem and progenitor cells as measured by flow cytometry and colony forming assays. With the milestone of E-CEL UVEC release for GLP material produced in a GMP facility achieved, AB-110 is now being tested towards completion of IND-enabling pre-clinical studies required by the US FDA prior to entry into clinical trials.