International Stem Cell Corp. (ISCO) specializes in the therapeutic applications of human stem cells and the development and commercialization of cell-based biomedical products. The company was the first to develop and perfect a new class of human stem cells called parthenogenetic stem cells, created from unfertilized human eggs. ISCO has a strong patent portfolio offering clean intellectual property and freedom to operate. The company's stem cells present superior immune matching capabilities and can be used in millions of people regardless of sex or racial background, with minimal expectation of immune rejection after transplantation.
The company's human stem cells have been shown to be as pluripotent as embryonic stem cells; however, their creation does not involve the destruction of a viable human embryo, which effectively sidesteps the controversy and ethical dilemmas associated with the use of human embryonic stem cells. In contrast to induced pluripotent stem cells, ISCO's stem cells do not involve manipulation of cells' genome, thereby avoiding potential safety and regulatory obstacles in clinical applications.
The company's scientists are currently focused on using its stem cells to treat severe unmet medical needs of the central nervous system (Parkinson's disease), the liver and the eye, where cell therapy has been clinically proven but is limited due to the unavailability of safe human cells. Once the technology has been clinically validated there are an essentially unlimited number of potential applications. Because of their immune-matching ability a relatively small number of these stem cell lines could offer the potential of producing the first true stem cell bank as a means of serving populations of different immune types across the globe.
In addition to its therapeutic focus, ISCO also provides a growing revenue stream through two wholly owned subsidiaries. Lifeline Cell Technology specializes in producing primary human cells and growth media for biological research, and Lifeline Skin Care, the company manufactures and markets advanced anti-aging skincare products utilizing the company's expertise in stem cell biology.
- New Stroke Program
- Growing Revenues - $7M
- Robust Pre-Clinical Pipeline
- Powerful Stem Cell Platform
- Broadly Applicable Platform
- IP Position in U.S., EU, Japan
- Near-term Catalysts in Parkinson's Disease
Stem cells are immortal cells that can both proliferate (divide) and change (differentiate) into more specialized cells such as skin, liver or blood cells. The most powerful stem cells are known as "pluripotent" because they have the potential to become any cell in the body. The first pluripotent stem cells to be studied were embryonic stem cells, but because creating embryonic stem cells involves the destruction of a fertilized human embryo, many people have ethical concerns about the use of such cells. Embryonic stem cells also suffer from the disadvantage of not immune matching most people requiring the use of immuno-suppression drugs to prevent tissue or cells derived from embryonic stem cells being rejected by the recipient.
The four most commonly used and described classes of stem cells are:
- Embryonic stem cells (embryonic SCs)
Induced pluripotent stem cells (iPS)
Adult stem cells (adult SCs)
Parthenogenetic stem cells - hpSCs
Parthenogenetic stem cells developed by ISCO are the only class of cells that meet all the criteria that are important when considering therapeutic applications.
ISCO's large patent portfolio includes stem cell creation and differentiation methods
ISCO protects its technology worldwide by filing patent applications covering specific pluripotent human parthenogenetic stem cells (hpSC) lines, methods to produce new hpSC lines, and various differentiation methods for research, therapeutic and commercial uses. The majority of ISCO's patents and licenses has been filed in the U.S. and internationally and covers most industrialized countries.
Stem Cell Bank
The world's largest collection of non-embryonic histocompatible human stem cells for research and commercial use.
ISCO's UniStemCell bank is the life science industry's first collection of non-embryonic histocompatible human stem cells available for research and commercial use. ISCO has developed a proprietary technique for creating histocompatible stem cells.
Human pluripotent stem cells from unfertilized eggs
The process, called parthenogenesis, uses unfertilized human eggs and results in the cells inheriting a duplicate set of human leukocyte antigen (HLA) genes. This significantly reduces the possibility of the derived cells being rejected by an individual's immune system making a single cell line suitable for treating millions of individuals. A relatively small number of such cell lines could be sufficient to provide "immune matched" cells to a large percentage of the world's population.
Bypasses ethical concerns
The stem cells are created by chemically stimulating the oocytes (eggs) to begin division. The oocytes are not fertilized and no viable embryo is created or destroyed.
Clinically superior for allogeneic therapy
Different activation techniques applied to human oocytes allow the creation of either HLA heterozygous human parthenogenetic stem cell lines (hpSC), which are exactly HLA-matched/ histocompatible with the oocyte donor, or HLA homozygous hpSC, which may be histocompatible with significant segments of the human population.
Can be used to treat millions of patients and many diseases
Normally donor tissue is screened for antigens in order to determine the degree of histocompatiblity with the recipient at the major histocompatibility complex (MHC). The human leukocyte antigen (HLA) system is the term used for the human MHC and represents antigens important for transplantation.
When used for transplant-based stem cell therapies, stem cells are likely to face the same HLA matching issues that limit solid organ allogeneic transplants and lead to immune rejection. The risk of rejection is proportional to the degree of disparity between donor and recipient cell-surface antigen-presenting proteins.
Matching donor and recipient tissue for HLA antigens greatly increases the likelihood of transplant survival. Parthenogenetic activation of human oocytes may be one way to produce histocompatible/HLA matched cells for cell-based therapy.
The ethical advantage of derivation from unfertilized oocytes combined with immunomatching advantage makes ISCO's stem cells a very promising source for cell-based therapy. This significantly reduces the possibility of the derived cells being rejected by an individual's immune system making a single cell line suitable for treating millions of individuals. A relatively small number of such cell lines could be sufficient to provide "immune matched" cells to a large percentage of the world's population.
Human parthenogenesis (unfertilized oocytes)
In the medium term, revenue could be generated from universal stem cell bank franchises across populations and scientists accessing these lines. This will provide additional validation of the hpSC lines as they will be tested and developed by others across a range of potential applications and, longer-term, will provide the company with royalty from sales of each successful, hpSC-derived cellular therapeutic.
Development of stem cell treatments for neurological disorders, diseases of the liver and blindness caused by corneal damage.
ISCO's scientists are focused on using this technology to treat severe diseases of the central nervous system, the liver and the eye where cell therapy has been clinically proven but treatment options are limited by the availability of safe human cells.
Broad Pipeline of Targets
Neural stem cells
We have used our multi-decade experience in human cell culture and our pioneering ethical pluripotent stem cell technology to create our first clinical product, human parthenogenetic neural stem cells, hpNSC. Neural stem cells are self-renewing multipotent cells that are precursors for the major cells of the central nervous system. ISCO's master cell bank (MCB) and working cell banks (WCB) of hpNSC are produced under cGMP conditions and consist of cryopreserved high purity hpNSCs derived using a highly-optimized, patented, chemically-defined differentiation process. To ensure the purity of the final product, the qualification process involves testing each cell bank for cell identity, potency, purity safety and sterility. For the proposed clinical trials, frozen aliquots of QC-released hpNSC from a WCB will be shipped to the trial site where the cell will be thawed and tested for viability before being used. All cells are produced at our Oceanside, California facility.
According to the Parkinson's Disease Foundation, an estimated seven to 10 million people worldwide live with Parkinson's disease, with as many as one million of those in the United States alone, more than the combined total of people diagnosed with multiple sclerosis, muscular dystrophy, and Lou Gehrig's disease. The total direct and indirect cost of Parkinson's disease is estimated to be nearly $25 billion per year in the United States alone.
Unlike conventional treatments for Parkinson's disease, our lead product candidate hpNSC works in a very different way. The ability of NSC's to both differentiate into dopaminergic neurons and express brain-protecting neurotrophic factors, offers a new approach to treating Parkinson's disease. We believe that a one-time transplant of hpNSCs into the mid-brain of Parkinson's patients, replacing the dead and dying neurons and offering protection to the remaining neurons, will alleviate the current symptoms and prevent further deterioration. Pre-clinical testing in mice, rats and primates support this theory.
The National Stroke Association lists stroke as a leading cause of death In the United States, killing nearly 130,000 people each year, and a leading cause of serious, long-term adult disability. Approximately 795,000 strokes occur each year taking a life approximately every four minutes. About 87% of strokes are ischemic. The estimated direct and indirect cost of stroke in the United States in 2010 is $73.7 billion.
During the acute phase of an ischemic stroke the only treatment option is to attempt to dissolve the blood clot causing the infarct. This can be effective if done within the first two to three hours post stroke. After this acute phase, treatment options primarily involve rehabilitation to improve cognitive and functional recovery.
There is pre-clinical evidence that NSC can reverse the functional deficits of a stroke up to several weeks after the initial event. ISCO will begin testing the highly-pure hpNSC product in a number of pre-clinical models of ischemic stroke.
ISCO has created and characterized stem cell-derived liver cells (CytoHep) from human parthenogenetic stem cells. Such cells can potentially be used to treat a number of liver diseases.
Metabolic Liver diseases
Crigler-Najjar syndrome is a serious inherited metabolic liver disease where the liver lacks a critical enzyme responsible for metabolizing the toxin bilarubin. The absence of this enzyme causes a build-up of bilarubin in the blood which can lead to brain and nerve damage. The most severe form of the syndrome can result in early death, however, it has been show clinically that transplanting liver cells into patient's liver can help alleviate the symptoms of the disease.
ISCO's R&D team have shown that stem cell-derived liver cells behave in a way that is very similar to hepatocytes and that implanting these stem cell-derived hepatocytes in pre-clinical animal models of Crigler-Najjar syndrome can have positive benefits.
ISCO scientists have developed methods to create human retinal epithelium (RPE) cells from parthenogenetic stem cells. These cells can be used to treat many diseases including age-related macular degeneration (AMD).
ISCO scientists were among the first scientists in the world to develop methods to create human corneal cells and whole corneal tissue from parthenogenetic stem cells. These cells and tissue can be used to treat many diseases including forms of corneal blindness.
Parkinson's Disease Program
Parkinson's disease is the second-most common neurodegenerative disease, affecting approximately 10 million people worldwide. While advances in research are underway, the disease continues to progress. Current medications only address the symptoms - there is currently no cure.
ISCO is the first company to treat Parkinson's disease with PSC-derived cells and in April 2014 reported that behavioral improvements have been observed after six months in the pre-clinical non-human primate (NHP) study of Parkinson's disease.
The company has conducted two safety studies in which human parthenogenetic neural stem cells (hpNSC) were transplanted into the brains of healthy animals, and a proof-of-principle study where the cells were transplanted into animals with induced Parkinson's disease symptoms. The studies show that hpNSCs migrated to the injured brain area, increased dopamine levels, protected and recovered neurons and improved the motor function. Additionally, at the conclusion of the 12 month non-human primate safety study, no evidence of teratoma formation or ectopic tissue was found in any animals that received the human cell transplants.
The U.S. Food and Drug Administration (FDA) has cleared ISCO's parthenogenetic stem cells, the starting material for the production of ISC-hpNSC, for clinical use.
In addition, ISCO has completed the required preclinical studies and in mid-2015plans to begin the phase 1/2a clinical study of the company's cell therapy for the treatment of Parkinson's disease in Australia. The company has formed an Australian subsidiary, Cyto Therapeutics Pty Ltd, to manage the regulatory submission to the Australian Therapeutics Goods Administration (TGA) and the up-and-coming clinical study. The submission includes non-clinical studies evaluating the safety and tolerability of the clinical product, ISC-hpNSC, in different animal species, including non-human primates, along with the chemistry and manufacturing controls and a summary of the clinical study design.
ISCO believes that its chemistry and manufacturing controls, which ensure that each batch of cells produced at the company's GMP facility in Oceanside, California, is subject to standardized quality control and assurance, meets both the US FDA and the Australian TGA standards.
A novel business model for the biotech industry; comprising revenue-generating subsidiary business units.
ISCO operates under a novel business model for the biotechnology industry. This self-sustaining model comprises subsidiary business units that generate revenue from the sale of products developed from scientific discoveries made by ISCO. Revenues are used to support further scientific research and development, as well as provide practical, short-term applications of the core technologies.
Lifeline Cell Technology
Lifeline specializes in the development and manufacture of purified primary human cells and optimized reagents for cell culture. Lifeline's scientists have been working in this important field of cell culture for over 20 years and developed many of the human cell systems and the quality standards that are used today in academic, government and pharmaceutical laboratories to study human disease. The company's team of scientists and quality control technicians continue to make new and improved human cell systems to meet the needs of a more demanding cell culture marketplace for primary human cells and are making breakthroughs in the development of human stem cell products.
- A vertically integrated primary human cell research products business
$2.3 billion biomedical products market (bcc research 2009)
More than 130 cells and optimized culture media products
Growing domestic sales force for branded products
International distributors in Asia and Europe
Lifeline Skin Care
While researching cures for diabetes and Parkinson's disease, a team of biotech scientists discovered a powerful compound for regenerating skin cells. This discovery has created a new era in anti-aging skin care products that rejuvenate your skin by actually helping to build new skin cells.
- Branded anti-aging stem cell skin care products
$800 million cosmeceutical market (Kline 2011)
Patented technology backed by clinical studies
Premium brand endorsed by dermatologists
International distributors and multiple sales channels
Award-winning performance boosted by revenue-generating subsidiaries
ISCO was awarded the designation of one of America's fastest growing companies as highlighted in Deloitte's 2014 Technology Fast 500™ list in recognition of the company's rate of growth in sales over the last several years.
ISCO reported $7.02 million in revenue for the year ended December 31, 2014, an increase of 14% compared to 2013. Lifeline Skin Care sales increased 9% while Lifeline Cell Technology sales grew 19%. Gross margin stable at 73%
Andrey Semechkin, PhD. - Chief Executive Officer and Co-Chairman of the Board
Dr. Andrey Semechkin is a specialist in system analysis, strategic planning and corporate management. He is a member of the Russian Academy of Sciences and has been Deputy Director of Institute of System Analysis since 2004. Professor Semechkin was awarded the Russian Government Award in Science and Technology in 2006 and has written several scientific books. He has more than 20 years of experience creating and managing businesses across different industries and scientific sectors.
Simon Craw, PhD. - Executive Vice President of Business Development
Dr. Simon Craw obtained his PhD in Chemistry from the University of Manchester and began his research career in Brazil at the University of Rio de Janeiro, followed by positions at the University of Sydney, Australia, before returning to the University of Manchester, England. He has more than 18 years of experience in the life science industry, including time at Novartis Pharmaceuticals and Astra-Zeneca followed by eight years at Merck Research Laboratories and five years at ACADIA Pharmaceuticals in San Diego, Calif. Dr. Craw is a frequent presenter at scientific and investment conferences and has a significant number of scientific publications. He is often featured in local and nation media discussing stem cells and regenerative medicine, including appearances on both National Public Radio and Fox News.
Jay Novak - Chief Financial Officer
Jay Novak has more than 18 years of experience in finance and accounting. He has served as director of finance since May 2012. From July 2011 to May 2012, he was senior manager, financial reporting. Prior to joining ISCO, Novak has served in various finance and accounting roles at several publically traded companies. His experience includes serving as associate director of finance at Nanogen, Inc., associate director of finance at Elan Pharmaceuticals, and assistant director of finance at Isis Pharmaceuticals, Inc. Novak is a certified public accountant, and began his career with Deloitte & Touche, LLP. He has received a B.S in Accountancy from California State University, Long Beach and an M.B.A. from University of California, Irvine.
Ruslan Semechkin, PhD. - Chief Scientific Officer
Dr. Ruslan Semechkin was trained in medical genetics, stem cell biology and international business administration and holds an MS degree from Faculty of Fundamental Medicine of Moscow State University. He earned his PhD degree in Physiology from Anokhin Research Institute of Normal Physiology, Russian Academy of Medical Sciences. Dr. Semechkin is a well-known speaker on stem cell biology, including the use of stem cells for neurology and skin regeneration. He has publications in the field of clinical and molecular biology, and is author of various patent applications.
Ruslan Semechkin, PhD - Chief Scientific Officer
Dr. Ruslan Semechkin was trained in medical genetics, embryology and stem cell biology. He holds a PhD in Human Physiology from the Russian Academy of Medical Sciences and has broad expertise in neuroscience, and was part of the team, along with scientists from the NYU Medical School that elucidated the physiological changes that occur in the brains of Parkinson's disease patients. Dr. Semechkin directs ISCO's R&D programs including stem cell derivation, differentiation and the pre-clinical and clinical evaluation of stem cell derived cells and tissue. He has developed a general method of deriving highly pure populations of neural stem cells and dopaminergic neurons from pluripotent stems cells that is novel, practical and suitable for use in a clinical setting. Dr. Semechkin is a well-known speaker on stem cell biology, including the use of stem cells for neurology and skin regeneration. He has more than 40 publications in the field of Parkinson's disease and stem cell biology and he is an active member of the American Academy of Neurology, the Society for Neuroscience and the International Society for Stem Cell Research.
Donna Queen - President of Lifeline Skin Care Inc.
Donna Queen has more than 20 years of experience as a marketing executive. Prior to joining ISCO, Queen was president and CEO of ZO SKIN HEALTH® by Zein Obagi, MD. Dr. Obagi is the dermatologist who created the original Obagi Nu-Derm skincare system, which has since become the leading physician-dispensed brand of anti-aging skincare. Earlier, Queen founded and led one of Virginia's largest advertising and marketing agencies, specializing in aesthetic and dermatological marketing and brand development.
Francisco Bustamante - President of Lifeline Cell Technology, LLC
Francisco Bustamante has more than 18 years of experience in operations of biotechnology companies. His experience includes senior management positions in the areas of manufacturing, procurement, planning, warehousing, distribution and project management. Bustamante has an excellent understanding of the manufacture and logistics of cell culture products, biological instruments, molecular biology kits and diagnostics. He has led key projects in the areas of manufacturing resource planning (MRP) systems implementation, ISO compliance and product development. His industry experience includes Clonetics, BioWhittaker (Cambrex), Digene and Meso Scale Diagnostics. Bustamante received his B.S. degree in Biology from the University of San Diego and his MBA degree from Frostburg State University. He has been with Lifeline Cell Technology since 2007.
Jeremy Hammond - Director of Quality Control
Jerry Hammond has more than 18 years of experience developing human cell-based products and the quality control systems critical for their manufacture. He created and implemented the quality control systems that were responsible for making Clonetics products world renown for consistency and reproducibility. These quality control systems are the standard for companies currently manufacturing human cell based products. Hammond is expert in the field of both serum-containing and serum-free media formulations and in the purification of human cells, fields critical for therapeutic applications of cell culture technology.
Glenn Sherman - Director of Chemistry Manufacturing and Controls (CMC)
Dr. Glenn Sherman has more than 20 years of experience in regulatory affairs. He worked as a primary microbiology reviewer at the U.S. FDA where he led pre-IND reviews in the Division of Antiviral Drug Products. After leaving the FDA Dr. Sherman held regulatory positions at Pfizer and Johnson & Johnson, where he was CMC regulatory lead for biologics products and successfully managed IND related activities for the biologics clinical, nonclinical and CMC teams. Dr. Sherman holds a PhD in Microbiology and Immunology from the University of North Carolina, Chapel Hill, NC.
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