StemCells, Inc. is engaged in the research, development, and commercialization of stem cell therapeutics and related enabling technologies for academia and industry. We believe that understanding cells and cell biology, and in particular stem cells, will play an increasingly important role in the understanding of human diseases and in the discovery of new medical therapies. Consequently, we are focused on (i) cellular medicine, or the use of stem and progenitor cells as the basis for novel therapeutics and therapies, and (ii) enabling technologies for stem cell research, or the use of cells and related technologies to enable stem cell-based research and drug discovery and development.
Our primary research and development efforts are focused on cellular medicine, where we seek to identify and develop stem and progenitor cells as potential therapeutic agents. We currently have two therapeutic product development programs: (i) our CNS Program, which is developing applications for HuCNS-SC® cells, our proprietary human neural stem cell product candidate, and (ii) our Liver Program, which is developing applications for our proprietary human liver engrafting cells. We estimate that degenerative conditions of the central nervous system (CNS) and the liver together currently affect more than 35 million people in the United States.1
In our CNS Program, we are in clinical development with our HuCNS-SC cells for disorders of the central nervous system, which includes the brain, spinal cord and eye. We have completed a Phase I clinical trial to evaluate the safety and preliminary efficacy of our HuCNS-SC cells as a treatment for infantile and late infantile neuronal ceroid lipofuscinosis (NCL), two forms of a group of disorders often referred to as Batten disease. NCL is fatal and there are currently no approved treatments for the disease. The data from our NCL trial showed that our HuCNS-SC cells were well tolerated, non-tumorigenic, and there was evidence of engraftment and long-term survival of the HuCNS-SC cells. In November 2009, we initiated a Phase I clinical trial of our HuCNS-SC cells in a second indication, Pelizeaus-Merzbacher Disease (PMD), a fatal myelination disorder in the brain. In February 2010, we enrolled and treated the first patient in our PMD trial, and we expect it will take 12-18 months to complete enrollment. In addition, our HuCNS-SC cells are in preclinical development for spinal cord injury and retinal disorders.
In our Liver Program, we are in preclinical development with our human liver engrafting cells (hLEC) to evaluate hLEC as a potential cellular therapy for a range of liver diseases. In September 2009, we received ethics committee approval to initiate a clinical study to evaluate hLEC as a potential cellular therapy for liver-based metabolic disorders. However, we have decided to defer initiating a clinical study of hLEC, pending additional improvements to our process of isolating and purifying hLEC.
In our enabling cell technologies programs, we are engaged in developing and commercializing applications of our technologies to enable stem cell-based research. We currently market a range of proprietary cell culture products under the SC Proven® brand, including iSTEM®, GS1-RTM, GS2-MTM, RHB-A®, RHB-Basal®, NDiff® N2B27, NDiff® 2 and 27 supplements, HEScGROTM, and ESGRO CompleteTMproprietary media.2 Academic and industrial laboratories conducting stem cell research need specialized cell culture products for the derivation, growth, maintenance and manipulation of stem cells, and as this type of research continues to grow, the market for such cell culture products will also continue to expand. In addition, the pharmaceutical industry has shown an increasing interest in the use of stem cell-based assays in its research and development activities. We are pursuing the development of our technologies, including technologies relating to embryonic stem cells, induced pluripotent stem (iPS) cells, and tissue-derived (adult) stem cells, as well as a gene insertion technology, for use in drug discovery and development. Several of the cell technologies and intellectual property related to our enabling cell technologies programs were acquired in April 2009 through our acquisition of substantially all of the operating assets and liabilities of Stem Cell Sciences Plc (SCS).
Our aim is to create a sustainable business based on our belief that understanding cells and cell biology will play an increasingly important role in life science research and in the discovery, development and implementation of new medical therapies. Our primary strategy is to identify multiple types of human stem and progenitor cells with therapeutic and commercial importance, to develop techniques and processes to purify these cells for direct transplant and to expand and bank these cells, to advance these cells into clinical development and ultimately, to commercialize them as cell-based therapeutic products.
The fundamental competencies required to execute this strategy are knowledge and expertise in cell biology, particularly stem cell biology, and a commitment to rigorous and robust research and development. We believe that these competencies are critical to identifying, characterizing and understanding cells with therapeutic potential and importance, and ultimately, that “good science makes for good medicine.”
Consequently, we have made significant investments in our research and development, clinical and regulatory, and cell processing and process development capabilities. Our management and staff have many years of experience in the stem cell field and in developing potential cell therapies. Two of the four human stem cells identified and characterized to date (the hematopoietic and neural stem cells) were discovered by scientists who are currently on our staff, and we believe we were the first company to receive authorization from the U.S. Food and Drug Administration (“FDA”) to conduct a clinical trial of a purified neural stem cell product candidate, as well as the first to complete such a clinical trial.
Many of our core competencies in cell biology have applicability beyond the development of therapeutic products. Therefore, another element of our business strategy is to leverage these core competencies to develop non-therapeutic applications for our cell technologies, which we believe represent nearer-term commercial opportunities. As scientific and medical research increasingly focuses on stem cells and cell biology, our technologies are expected to have utility as tools to help enable this research. We currently market specialized cell culture products through our SC Proven product line and are seeking to develop and commercialize applications of our technologies for use in stem cell-based assays.
Further, a key element of our business strategy is to obtain patent protection for the compositions, processes and uses of multiple types of cells, as well as for those technologies that appear applicable and useful to enable cell-based research. We believe that patent protection will be available to the first to identify and isolate any of the finite number of different types of human stem and progenitor cells, and the first to define methods to culture such cells, making the commercial development of cell-based therapeutics and enabling applications financially feasible. In addition to discovering and developing technologies in-house, we have obtained from various academic and commercial institutions rights to certain inventions relating to stem and progenitor cells, cell culture media, and technologies to reprogram, isolate and manipulate cells. We expect to continue to expand our search for, and to seek to acquire rights from third parties relating to, new stem and progenitor cells and cell technologies.
As of December 31, 2009, we had 75 full-time employees, 20 of whom have Ph.D., M.D. or D.V.M. degrees. 59 full-time employees work in research and development and laboratory support services. No employees are covered by collective bargaining agreements. We consider our employee relations in general to be good.