We believe that our proprietary technology and the cell populations we produce have several potential advantages, including the following:
- Ability to select ALDHbr cells across multiple stem cell types.
We believe our ability to isolate ALDHbr cells and develop product candidates based on these cells is a key advantage. Based on preclinical studies and our clinical trials to date, we believe our ALDHbr cell populations have the ability to promote angiogenesis and improved vascular function, which are critical to treating cardiovascular disease. We believe ALDHbr cells are superior to unsorted cell populations or cell populations lacking ALDHbr cells altogether in this regard. We hypothesize that other bone marrow cells that do not exhibithigh levels of ALDH impede the ability of ALDHbr cells to interact with ischemic tissue in therapeutically effective ways. In addition, we believe the heterogeneous mix of different types of stem cells contained in the ALDHbr populations we produce provides advantages compared to homogeneous cell populations containing only one particular type of stem cell because heterogeneous populations may promote tissue repair in a variety of ways.
- Broad potential therapeutic opportunity.
We believe ALDHbr cells have broad potential applicability in promoting the regeneration of multiple types of cells, blood vessels and tissues. We believe this enhances the long-term potential of our technology to address a number of different therapeutic areas, which we might pursue either by ourselves or with collaborative partners.
- Specific applicability to unmet medical needs in the cardiovascular field.
Preclinical research suggeststhat ALDHbr cells may specifically migrate to sites of ischemic damage and induce the formation of new blood vessels at those sites. In our clinical trials to date, we have also observed evidence of improved perfusion in ischemic tissue. We believe that the indications our product candidates are intended to address—critical limb ischemia, ischemic heart failure and ischemic stroke, in each case for which patients have few or no treatment options—represent an aggregate annual market opportunity of $11.5 billion in the United States.
- Autologous cells.
Autologous therapies use cells derived from the patient receiving the therapy, while allogeneic therapies use cells derived from a separate donor or donors. Our product candidates are based entirely on autologous cells collected from the patient’s own bone marrow, which we believe minimizes the risk of potential rejection of the injected cells in the patient. To date, the only cell therapies approved by the FDA have been based on autologous cells.
Because our manufacturing process does not require any culturing or the addition of any growth factor, we are able to produce well-characterized stem cell populations with a high level of purity and a consistent set of physical and chemical characteristics. We believe that our ability to produce well-characterized populations enhances our ability to meet regulatory requirements relating to safety and efficacy. In addition, our rapid turnaround time—typically 36 hours once we have received the bone marrow—is shorter than many other treatments involving autologous cells and allows for advantages in the scheduling of patient treatments. We believe that our manufacturing process is scalable to support commercialization, a key factor that may be considered by potential collaborative partners.