SCSI Component Companies

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The Stem Cell Stock Index provides a quantitative lens through which America's leading stem cell therapeutic companies (listed at left) can be meaningfully compared and contrasted, as we do in our report, The State of the American Stem Cell Industry In 2012: Publicly Traded U.S. Stem Cell Companies By the Numbers. The index is currently comprised of eighteen leading companies, fourteen of whose business and technical performances in 2012 are the subjects of that report.


Aastrom Biosciences Inc. (NASDAQ:ASTM)
www.aastrom.com

Michigan-based Aastrom Biosciences develops patient-specific (autologous), expanded multicellular therapies for use in the treatment of severe, chronic ischemic cardiovascular diseases such as critical limb ischemia and dilated cardiomyopathy. Its lead candidate, ixmyelocel-T, is manufactured by Aastrom by expanding cells from a patient’s own bone marrow.

Clinical trials by Aastrom Biosciences currently tracked by the SCSI Clinical Trials Monitor include:

Recent headlines for Aastrom Biosciences:

Recent Organelles essays for Aastrom Biosciences:

Aastrom earns special mention in sections of our industry analysis including Clinical Trials and Market Snapshots.


Advanced Cell Technology, Inc. (OTCBB:ACTC)
www.advancedcell.com

Massachusetts-based Advanced Cell Technology (ACT) has been the subject of frequent controversies and lawsuits. Its lead therapeutic candidates are cells differentiated from human embryonic stem cells, including retinal pigment epithelial cells for treatment of Stargardt’s macular dystrophy and age-related macular degeneration, myoblasts for the treatment of heart failure, and hemangioblasts for vascular repair.

Clinical trials by Advanced Cell Technology currently tracked by the SCSI Clinical Trials Monitor include:

Recent headlines for Advanced Cell Technology:

Recent Organelles essays for Advanced Cell Technology:

Advanced Cell Technology earns special mention in sections of our industry analysis including Grant Revenue, Leadership Compensation, Clinical Trials, and Market Snapshots.


Athersys, Inc. (NASDAQ:ATHX)
www.athersys.com

Ohio-based Athersys' lead candidate, MultiStem®, is an allogeneic stem cell product derived from the bone marrow of healthy donors and expanded in a proprietary process. MultiStem has been evaluated in two completed clinical trials, for heart attack and graft versus host disease, and is currently in ongoing trials: one for inflammatory bowel disease with partner Pfizer Inc., and another for neurological damage from stroke.

Clinical trials by Athersys, Inc. currently tracked by the SCSI Clinical Trials Monitor include:

Recent headlines for Athersys, Inc.:

Recent Organelles essays for Athersys, Inc.:

Athersys earns special mention in sections of our industry analysis including Institutional Investors, Dilution, Clinical Trials, What Moved The Market in 2012, and Market Snapshots.


BioTime, Inc. (NYSEMKT:BTX)
www.biotimeinc.com/

BioTime is based in California. Unlike most other SCSI component companies, it is heavily focused on near-term commercial opportunities from research products, including embryonic stem and other cell lines, culture media, and hydrogels. BioTime states that its long-term strategic goal is to develop regenerative therapies for age-related degenerative diseases.

The SCSI Clinical Trials Monitor does not currently include any clinical trials sponsored by BioTime.

Recent headlines for BioTime:

Recent Organelles essays for BioTime:

BioTime earns special mention in sections of our industry analysis including SCSI Index Performance, Institutional Investors, Dilution, and Market Snapshots.


bluebird bio, Inc. (NASDAQ:BLUE)
www.bluebirdbio.com

bluebird bio (the company uses all lower-case letters in its name) is based in Cambridge, MA, with additional facilities in San Bruno, CA and Paris, France. With the closing of its initial public offering in June, 2013, bluebird bio became the sixteenth component company of the SCSI: Stem Cell Stock Index.

bluebird describes itself as a "clinical-stage biotechnology company focused on transforming the lives of patients with severe genetic and orphan diseases using gene therapy,", not as a stem cell company per se. Nevertheless, the company's gene therapy technology critically depends on autologous (the patient's own) hematopoietic stem cells (HSCs), which it genetically re-engineers in vitro by transfecting them with functional copies of the patient's mutated (non-functional) disease-causing gene before returning them to the patient. These re-engineered stem cells can then, the company believes, reproduce and form normally functioning blood cells throughout the patient's life, thus curing the disease. In short, bluebird employs stem cells as genetically engineered delivery vehicles for gene therapy, rather than as therapeutics in their own right. The company currently focuses its development efforts on the genetic diseases cerebral adrenoleukodystrophy (CCALD), β-thalassemia, and sickle cell disease.

bluebird's market cap of $648 million in mid-August 2013 (when it joined the SCSI) made this newcomer by far the highest valued SCSI component company (ahead of #2 Osiris Therapeutics at $345 million).

Clinical trials by bluebird bio currently tracked by the SCSI Clinical Trials Monitor include:

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Recent Organelles essays for bluebird bio:


BrainStorm Cell Therapeutics Inc. (OTCBB:BCLI)
www.brainstorm-cell.com

BrainStorm is incorporated in the U.S. but based in Israel. Its lead candidate, NurOwn™, is produced from each patient’s own bone marrow mesenchymal stem cells expanded and differentiated in vitro in a proprietary process to create neurotrophic factor-secreting cells that the company believes may maintain, preserve and possibly restore the damaged neurons of patients with neurodegenerative diseases such as amyotrophic lateral sclerosis.

Clinical trials by BrainStorm Cell Therapeutics currently tracked by the SCSI Clinical Trials Monitor include:

Recent headlines for BrainStorm Cell Therapeutics:

Recent Organelles essays for BrainStorm Cell Therapeutics:

BrainStorm earns special mention in sections of our industry analysis including The SCSI Fourteen, Institutional Investors, and Leadership Compensation.


Capricor Therapeutics Inc. (OTCMKTS:CAPR)
www.capricor.com

California-based Capricor went public in November, 2013 via a reverse merger with Nile Therapeutics and an immediate 1-for-50 reverse stock split. It initiated trading under the symbol NLTXD, switching to CAPR on December 20, 2012. Capricor develops cardiosphere-derived cells (also referred to as cardiac stem cells). CAP-1002, its lead product candidate, is a proprietary allogeneic adult stem cell therapy for the treatment of heart damage. The product is derived from donor heart tissue. The cells are expanded in the laboratory using a specialized process and then introduced directly into a patient’s heart via infusion into a coronary artery using standard cardiac catheterization techniques. CAP-1002 is currently in a Phase 1/2 clinical trial, named ALLSTAR, for anterior myocardial infarction and ischemic left ventricular dysfunction. In January 2014 Capricor entered into a manufacturing collaboration for CAP-1002 with Janssen Biotech, a Johnson & Johnson company, earning Capricor $12.5 million up front and providing Janssen an option to license CAP-1002 for a potential $325 million plus royalties.

Clinical trials by Capricor Therapeutics currently tracked by the SCSI Clinical Trials Monitor include:

Recent headlines for Capricor Therapeutics:

Recent Organelles essays for Capricor Therapeutics:


Cellular Dynamics International, Inc. (NASDAQ:ICEL)
www.cellulardynamics.com

The newest component of the SCSI index (launching its $46 million IPO and beginning trading on the NASDAQ exchange on July 25, 2013), Cellular Dynamics International (CDI) is the only SCSI company with an exclusive focus on stem cells as research reagents (rather than as therapeutics). CDI pioneered the commercialization of cardiomyocytes (heart cells) and hepatocytes (liver cells) derived in vitro from human induced pluripotent stem cells (iPSCs), which it sells primarily for pharmaceutical drug discovery applications.

The SCSI Clinical Trials Monitor does not currently include any clinical trials sponsored by Cellular Dynamics International.

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Recent Organelles essays for Cellular Dynamics International:


Cytomedix, Inc. (OTCBB:CMXI)
www.cytomedix.com

In 2012 the company entered the stem cell therapeutics industry with its acquisition of North Carolina-based Aldagen, but in September of 2013 it announced its intention to shutter its stem cell development programs, which it is currently seeking to divest. Aldagen’s proprietary technology separates, from allogeneic or autologous cord blood or bone marrow, progenitor cells expressing high levels of aldehyde dehydrogenase. The company believes these are “biologically instructive” cells with the potential to promote the repair and regeneration of multiple types of cells and tissues, including blood vessels.

Clinical trials by Cytomedix currently tracked by the SCSI Clinical Trials Monitor include:

Recent headlines for Cytomedix:

Recent Organelles essays for Cytomedix:

Cytomedix earns special mention in sections of our industry analysis including The SCSI Fourteen, Dilution, Grant Revenue, Leadership Compensation, Clinical Trials, and Market Snapshots.


Cytori Therapeutics Inc. (NASDAQ:CYTX)
www.cytori.com

California-based Cytori's key product, the Celution® System, is a medical device that automates the extraction and preparation of clinical grade adipose-derived stem cells (a preparation the company terms “adipose-derived regenerative cells,” or ADRCs), at the point-of-care, from autologous liposuction aspirates. The most commercially advanced indication for the Celution System is breast reconstruction, but other therapeutic development activities focus on the use of Celution-prepared ADRCs to treat cardiovascular disease and repair soft tissue defects.

Clinical trials by Cytori Therapeutics currently tracked by the SCSI Clinical Trials Monitor include:

Recent headlines for Cytori Therapeutics:

Recent Organelles essays for Cytori Therapeutics:

Cytori earns special mention in sections of our industry analysis including The SCSI Fourteen, SCSI Index Performance, Institutional Investors, Dilution, Grant Revenue, Clinical Trials, What Moved The Market in 2012, and Market Snapshots.


Fate Therapeutics, Inc. (NASDAQ:FATE)
fatetherapeutics.com

Based in California, Fate is unique among SCSI component companies in that it develops molecular (rather than cellular) therapies, focusing on biological drugs that influence the behavior of stem cells. With the closing of its IPO in October, 2013, Fate became the 17th component company of the SCSI index.

Fate's lead candidate, ProHema, is intended to modulate the behavior of hematopoietic stem cells (HSCs) of umbilical cord blood used to reconstitute the immune systems of cancer patients undergoing myeloablative therapy, purportedly enhancing the homing, or migration, of HSCs to the recipient's bone marrow niche as well as increasing HSC proliferation and survival, thus speeding the transplant recipient's recovery of a working immune system and reducing the risk of life-threatening infections (a common complication of myeloablative therapy). ProHema is the company's proprietary version of the natural signaling molecule, prostaglandin E2, and is currently in one Phase 1 and two Phase 2 clinical trials sponsored by Fate. The company is also pursuing a pre-clinical program developing a natural human protein, Wnt7a, to promote the repair of muscle damage by stimulating the development of new muscle cells from skeletal muscle satellite stem cells, for treatment of muscular dystrophies.

Clinical trials by Fate Therapeutics currently tracked by the SCSI Clinical Trials Monitor include:

Recent headlines for Fate Therapeutics:

Recent Organelles essays for Fate Therapeutics:


International Stem Cell Corp. (OTCMKTS:ISCO)
www.internationalstemcell.com

International Stem Cell’s lead research products and pre-clinical therapeutic candidates are termed by it “human parthenogenetic stem cells” (“hpSCs”). The company believes these cells to be functional equivalents of embryonic stem cells. Its preclinical therapeutic development programs are exploring the use of cells produced from hpSCs for indications including liver disease, diabetes, Parkinson’s disease, and ocular diseases. International Stem Cell’s wholly-owned subsidiary, Lifeline Cell Technology, markets research reagents including primary cells, stem cells, and culture media.

The SCSI Clinical Trials Monitor does not currently include any clinical trials sponsored by International Stem Cell.

Recent headlines for International Stem Cell:

Recent Organelles essays for International Stem Cell:

International Stem Cell earns special mention in sections of our industry analysis including The SCSI Fourteen and SCSI Index Performance.


NeoStem Inc. (NYSEMKT:NBS)
www.neostem.com

In 2011 this New York-based company acquired Progenitor Cell Therapy, a GMP-compliant contract manufacturing organization for cell-based therapeutics, and Amorcyte, engaged in developing an autologous mesenchymal stem cell therapy for cardiovascular disease. Through its subsidiary, NeoStem Family Storage, LLC, the company also offers stem cell banking services in the U.S.

Clinical trials by NeoStem currently tracked by the SCSI Clinical Trials Monitor include:

Recent headlines for NeoStem:

Recent Organelles essays for NeoStem:


Neuralstem, Inc. (NYSEMKT:CUR)
www.neuralstem.com

Maryland-based Neuralstem's allogeneic neural stem cells are sourced from human brain or spinal tissue and expanded in vitro in a proprietary process. These cells are currently in Phase 1 clinical trials for amyotrophic lateral sclerosis and spinal cord injury.

Clinical trials by Neuralstem Inc. currently tracked by the SCSI Clinical Trials Monitor include:

Recent headlines for Neuralstem Inc.:

Recent Organelles essays for Neuralstem Inc.:

Neuralstem earns special mention in sections of our industry analysis including Dilution and Market Snapshots.


Osiris Therapeutics, Inc. (NASDAQ:OSIR)
www.osiris.com

This Maryland-based company has two expanded allogeneic MSC-based products in clinical trials: Chondrogen, a preparation of adult MSCs from bone marrow formulated for direct injection into the knee for regeneration of meniscus and prevention of osteoarthritis, and Prochymal, a preparation of adult MSCs from bone marrow for inflammatory, autoimmune and cardiovascular indications. In 2012 Osiris's Prochymal became the first stem cell therapeutic ever to receive marketing approval, by Canada and New Zealand for the treatment of graft-versus-host disease in children.

Clinical trials by Osiris Therapeutics currently tracked by the SCSI Clinical Trials Monitor include:

Recent headlines for Osiris Therapeutics:

Recent Organelles essays for Osiris Therapeutics:

Osiris earns special mention in sections of our industry analysis including The SCSI Fourteen, SCSI Index Performance, Institutional Investors, Dilution, Leadership Compensation, and Clinical Trials.


Pluristem Therapeutics Inc. (NASDAQ:PSTI)
www.pluristem.com

U.S. incorporated (but Israel-based) Pluristem’s candidate therapeutic cells are termed by it “placental expanded cells” or “PLX”: allogeneic expanded human MSCs derived from human placentas. The cells are manufactured by Pluristem using proprietary processes. Its lead product candidate, PLX-PAD, is being developed to treat critical limb ischemia, intermittent claudication, and Buerger's disease resulting from peripheral artery disease.

Clinical trials by Pluristem Ltd. currently tracked by the SCSI Clinical Trials Monitor include:

Recent headlines for Pluristem Ltd.:

Recent Organelles essays for Pluristem Ltd.:

Pluristem earns special mention in sections of our industry analysis including Clinical Trials.


StemCells Inc. (NASDAQ:STEM)
www.stemcellsinc.com

The company's strategy is to generate near-term revenue by marketing stem cell-related research reagents including proprietary cell culture products, antibodies, and cell lines, while pursuing a longer-term therapeutic development strategy. The latter aims to develop expanded allogeneic neuronal stem cells sourced from human fetal or adult nervous tissue (which StemCells terms HuCNS-SC® cells) for neurological indications including Pelizeaus-Merzbacher disease, Alzheimer’s disease, spinal cord injury, and macular degeneration.

Clinical trials by StemCells, Inc. currently tracked by the SCSI Clinical Trials Monitor include:

Recent headlines for StemCells, Inc.:

Recent Organelles essays for StemCells, Inc.:

StemCells earns special mention in sections of our industry analysis including SCSI Index Performance, Institutional Investors, Dilution, Grant Revenue, and Market Snapshots.


ThermoGenesis Corp. (NASDAQ:KOOL)
www.thermogenesis.com

The only SCSI component company with no therapeutic development program, ThermoGenesis manufactures and markets devices and disposable tools for the processing, storage and administration of cell and tissue therapies used in the practice of regenerative medicine. In July of 2013 the company announced its intention to merge with unprofitable, little-known, and privately held TotipotentRx, a provider of stem cell treatments in India employing the company's products, which would convey to TotipotentRx's shareholders 43% of the resulting company, to be re-named Cesca Therapeutics. The merger proposal -- which has proven highly controversial -- is to be voted on by Thermogenesis shareholders on February 13, 2014.

Clinical trials by Thermogenesis Corp. currently tracked by the SCSI Clinical Trials Monitor include:

Recent headlines for Thermogenesis Corp.:

Recent Organelles essays for Thermogenesis Corp.:


Non-SCSI Component Companies

Stem cell-focused companies that are not components of the SCSI, but which nonetheless earned special mention in our industry analysis include Celltex, Baxter International, IntelliCell BioSciences, Bioheart, and TiGenix.