Core A

HICTF & CVPF

Core A, located at both the Human Islet and Cellular Transplantation Facility (HICTF) at UCSF and the Clinical Cell and Vaccine Production Facility (CVPF) at the University of Pennsylvania will serve as the translational ‘bench to bedside’ Good Manufacturing Practice resource in the development of clinical grade Tregs for treatment of T1D.

  1. To perform the clinical trial scale-up, validation and qualification necessary to support the first clinical trials of Tregs in T1D. This includes qualification of a clinical grade method of Treg enrichment/separation from an apheresis product, optimization of clinical scale in vitro expansion, the development of a potency assay, and stability/shipping validation.
  2. To generate, cryopreserve and perform testing on a Master Cell Bank of K562-based artificial Antigen Presenting Cells (aAPC). The Master Cell Bank, produced from cells optimized during the studies proposed in Project #2, will then be used to expand Tregs in support of second generation clinical trials of Tregs for treatment of T1D.
  3. To perform cGMP Treg isolation and expansion and to serve as the cell and tissue repository in support of the clinical trials described in this application. To support Core B in the development and annual reporting requirements of the Investigational New Drug Application to the FDA for the clinical trials described in this application.

Translation of experimental cell therapies from concept through to clinical study demands a unique combination of specialized scientific, technical and regulatory expertise. This expertise must be accompanied by a dedicated infrastructure that meets or exceeds the rigorous requirements and regulatory guidelines necessary to ensure patient safety, particularly in early trials. The University of Pennsylvania CVPF and UCSF HICTF Are therefore uniquely and ideally suited to perform core functions in support of the translational and clinical research plans of the JDRF Collaborative Center for Cell Therapy. The CVPF has extensive experience in cell manipulation, growth, and transduction of lymphocytes and other immune cells as well as adherent cell lines, which combined with the increasing competence of the HICTF and the Blood Systems Research Institute (BSRI) in San Francisco at UCSF guarantees a robust set of facilities to accomplish the aims in this application. This experience combined with an extensive quality management program that has been codified in Standard Operating Procedures allows the Facilities to quickly and safely adapt new technologies to clinical trials.

 
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