Work package 5: Cell PouchTM implantation, BOEC cell transplantation and preclinical studies
Leader: Dr. Philip Toleikis, Sernova Corp.
Sernova Corp’s main objective is to demonstrate preclinical safety and efficacy of a haemophilia A patient’s blood cells that have been corrected to express Factor VIII and transplanted within Sernova Corp’s manufactured Cell Pouch™ device. The objective will be completed in a haemophilia A animal model and the information will become part of the regulatory documentation for a human clinical study to treat haemophilia A patients.
Description of the tasks
To fulfil the objective, Sernova Corp will complete the following tasks:
Task 1: Manufacture, package and sterilization of Cell Pouches™ to Good Manufacturing Practice (GMP) specifications for the preclinical animal studies.
Task 2: Receive the human Blood Outgrowth Endothelial Cells (BOECs) Factor VIII expressing product, shipped from Partner 4 UNILO to Sernova, and test the cells to ensure they meet the criteria for preclinical evaluation.
Task 3: Complete human BOEC Factor VIII/Cell Pouch™ cell dosing and engraftment studies and use this information to design a safety/efficacy study of GMP human BOEC Factor VIII expressing cells in the Cell Pouch™.
Task 4: Conduct safety and efficacy evaluation of the GMP human BOEC Factor VIII expressing cells in the GMP manufactured Cell Pouch™ using an established haemophilia A animal model (provided by Partner 3 UPO).
Upon completion of the tasks, Sernova will use this information in preparation of regulatory documentation for a future human clinical study which includes an “Instruction for Use” document with knowledge transfer of the product to be used by physicians to treat Haemophilia A patients.