We are developing MultiStem®, a proprietary stem cell product for the treatment of multiple distinct diseases and conditions in the cardiovascular, neurological, inflammatory and immune disease areas. MultiStem is a biologic product that is manufactured from human stem cells obtained from adult bone marrow or other nonembryonic tissue sources. Unlike other cell types, after isolation from a qualified donor MultiStem may be expanded on a large scale for future clinical use and stored in frozen form until needed. Cells obtained from a single donor require no genetic modification and may be used to produce banks yielding hundreds of thousands to millions of doses of MultiStem – an amount far greater than other stem cell types can achieve. Each bank is extensively characterized to ensure product consistency and safety.
MultiStem consists of a special class of human stem cells that have the ability to express a range of therapeutically relevant proteins and other factors, as well as form multiple cell types. Factors expressed by MultiStem have the potential to deliver a therapeutic benefit in several ways, such as reducing inflammation, protecting damaged or injured tissue, and enhancing the formation of new blood vessels in regions of ischemic injury. These cells exhibit a drug-like profile in that they act primarily through the production of multiple factors that regulate the immune system, protect damaged or injured cells, promote tissue repair and healing, and the cells are subsequently cleared from the body over time.
During several years of preclinical work, MultiStem has demonstrated the potential to address each of the fundamental limitations observed with traditional bone marrow or hematopoietic stem cell transplants. These limitations include the historical requirement for tissue matching between donor and patient, the typical need for one donor for each patient (a reflection of the inability to expand cells in a controlled and reproducible manner), frequent use of immune suppressive drugs to avoid rejection or immune system complications, and a range of other potential safety issues.
We believe that MultiStem represents a potential best-in-class stem cell therapy because it exhibits each of the following characteristics based on research and development to date: (1) it may be produced on an industrial scale, in a well validated and reproducible manner; (2) it may be administered without tissue matching, making it analogous to type O blood; (3) it exhibits a consistent safety profile; and (4) it appears capable of delivering therapeutic benefit through multiple mechanisms of action. Based upon work that we and independent collaborators have conducted over the past several years, we believe that MultiStem has the potential to treat a range of disease indications, including ischemic injury and cardiovascular disease, certain neurological diseases, autoimmune disease, transplant support (including in oncology patients), and a range of orphan disease indications.