Cellular Dynamics International, Inc. (CDI) (Nasdaq:ICEL) today announced that it has manufactured, under current Good Manufacturing Practices (cGMP), stem cell lines from two HLA “superdonors.” HLA superdonors are individuals whose genetic HLA (human leukocyte antigens, or HLA) profiles make their cells or tissues more compatible for donation to unrelated patients—a concern that arises with organ or tissue transplantation and potential rejection. As the first announced HLA superdonor master cell bank in the world, and the first produced under cGMP, these cell lines will enable a new area of cell therapy research using HLA matching.
CDI now has two HLA superdonor cell lines, providing a partial HLA match to 19 percent of the U.S. population. The Company plans to continue to expand this master stem cell bank with the collection of additional donor cell lines to cover 95 percent of the U.S. population.
CDI manufactured the HLA superdonor cell lines from blood samples collected from eligible anonymous donors. These donors consented to broad commercial use of their donated samples, including research and therapeutic applications. The induced pluripotent stem cell (iPSC) lines made from the donor samples are pluripotent, meaning they can be used to produce virtually any cell type in the human body.
cGMP conditions are required for the use of cells as a cellular therapy. CDI’s cGMP HLA superdonor stem cell lines can be used to manufacture tissue cells for potential cell therapy and organ rehabilitation. CDI currently manufactures human cells derived from iPSCs for more than 12 cell types, including brain, heart, liver, and blood vessel cells, under the iCell® brand name.
CDI’s HLA superdonor cell lines will be available, including use for developing cellular therapies within a clinical setting.
The HLA superdonor iPSC line was manufactured using CDI’s proprietary episomal reprogramming method. This non-integrating methodology is expected to alleviate major safety concerns over the potential use of iPSC-derived tissue cells in cellular therapies and removes a significant barrier for applying the technology in a clinical setting.
HLAs allow the immune system to distinguish “self” from “non-self” and are a key part of the match made in tissue and organ transplants.
Current alternatives for tissue or organ transplants include autologous, allogeneic, and partial match, or HLA superdonor options: CDI’s technology enables the autologous, personalized medicine approach, and the Company already has a contract with the National Eye Institute whereby Retinal Pigment Epithelial (RPE) cells will be manufactured from individuals suffering from dry age-related macular degeneration (AMD).
Allogeneic transplants have low or poor matching qualities between the donated tissue and the patient, thus resulting, too often, in rejection.
The HLA superdonor approach presents a partial HLA match that has been shown to be beneficial in organ transplants. With CDI’s proprietary process for mass producing human cells, in this case the HLA superdonor iPSCs and differentiated cell types made from these iPSCs, the Company has the ability to be the primary driver in this business.