RE:RE:RE:RE:busy times here...Bio its been in multiple MD&S's
In summary, the following developments have been achieved by the Consortium:
In blood donated from patients with Hemophilia A, blood endothelial outgrowth cells to be corrected for the Factor VIII gene were isolated and grown successfully in a specialized Good Manufacturing Process (cGMP) compliant medium developed by the Consortium.
Using a human Factor VIII gene insertion technique, the cells were corrected and confirmed to produce Factor VIII.
A preliminary experiment showed these cells could release Factor VIII in the blood over time and improve blood clotting in an animal model of Hemophilia A, in preparation for transplant into the Cell Pouch.
The corrected cells were proven to be successfully replicated through a production scale-up process. Following amplification, these cells maintained their normal healthy behaviour in producing Factor VIII. Additional safety metrics were achieved using established tests.
The cells were then cryopreserved and shipped from the European partners to Sernova in North America where they were shown to remain healthy through quality control testing in preparation for transplantation.
The Cell Pouch manufactured under cGMP, and following implantation in the Hemophilia A animal model showed development of vascularized chambers suitable to receive the corrected cells.
Following transplantation into the Cell Pouch in a Hemophilia A animal model, the patient’s Factor VIII corrected cells survived at three months (the duration of the study).
Initial results showed Factor VIII released from the cells in the Cell Pouch was detected in blood and notably, showed improved clotting when compared to the Hemophilia A animal control which did not receive human corrected cells.
The steps of the cell production process were documented towards development of the cGMP manufacturing process for the corrected cells for future clinical use. An Instructions-for-Use document was also developed for implantation of the cGMP Cell Pouch, and transplantation of patient corrected Factor VIII producing cells applicable for future human testing in patients with Hemophilia A.