** Listed alphabetically by company name, not in order of presentation. Please see the meeting agenda for presentation times.
ADERANS RESEARCH INSTITUTE
Aderans Research (ARI), headquartered in Marietta GA, is a leading force in the field of hair regeneration, pioneering innovative cell-based solutions to the problems of pattern hair loss that affect millions of people around the world. ARI is pioneering one of the most hopeful paths of research in the field, a method of cellular hair regeneration referred, namely the Ji Gami™ process. By rejuvenating hair-producing follicles, hair regeneration can generate an unlimited number of hairs from an individual’s own hair cells.
Presenter: Ken Washenik, M.D., Ph.D., CEO, Aderans Research Institute
ADVANCED CELL TECHNOLOGY
Advanced Cell Technology (“ACT”; ACTC.OB), is a biotechnology company developing cellular therapies for treating diseases that impact millions of people worldwide. Helmed by Chairman and CEO Gary Rabin, ACT is currently conducting the only human embryonic stem cell (hESC)-based clinical trials: two Phase I/II trials in the US and one in Europe. Initial results were published earlier this year in The Lancet. ACT is also focused on Hemangioblast platform for treating blood and cardiovascular diseases, and its mesenchymal stem cell program. ACT’s patented “embryo-safe” technique for deriving hESCs without harming embryos was documented in Nature.
Presenter: Gary Rabin, Chairman & CEO, Advanced Cell Technology
AlloSource, a non-profit organization, offers more than 200 types of precise bone, skin, soft-tissue and custom-machined allografts for use in life-saving and life-enhancing medical procedures. The world’s leader in fresh cartilage tissue and skin allografts, the company developed a cleansing process for fresh tissue grafts that does not destroy live cells. Most recently, AlloSource created a technique to recover mesenchymal stem cells from cadaveric adipose tissue, which led to the development of its own stem cell product, AlloStem® Stem Cell Bone Growth Substitute. AlloSource is registered with the FDA, accredited by the American Association of Tissue Banks and is compliant with all applicable state regulations and with the ISO 9001:2008 standard.
Presenter: Kevin Cmunt, Executive Vice President, AlloSource
Athersys (NASDAQ: ATHX) is an established leader in the emerging field of regenerative medicine, and currently has 5 clinical stage programs focused on treating significant unmet medical needs. The Company is developing its MultiStem® cell therapy product for treating disease indications in the cardiovascular, neurological, inflammatory and immune disease areas, among others. Current clinical programs include administration of MultiStem for treating inflammatory bowel disease (IBD) – partnered with Pfizer (in Phase 2), ischemic stroke (in Phase 2), for the prevention of graft-versus-host disease (Phase 2/3 pending), and for treating damage from acute myocardial infarction (Phase 2 pending).
Presenter: Gil Van Bokkelen, Ph.D., Chairman and CEO, Athersys
Capricor’s cardiac-derived stem cells (CDCs) represent a novel treatment to repair the heart after muscle loss following large heart attacks. This is accomplished through the regeneration of heart muscle and the shrinking of scar tissue. The CADUCEUS trial, which utilized Capricor’s CDCs, showed an approximate 50% reduction of scar size and 50% more viable heart muscle one-year post heart attack. This fall, Capricor plans to begin enrollment of ALLSTAR, a Phase I/II FDA approved study using allogeneic CDCs to treat patients 30-days to 1-year after a large heart attack. The goal is to show a relative reduction in scar tissue.
Presenter: Linda Marbán, Ph.D., CEO, Capricor
CEDARS-SINAI REGENERATIVE MEDICINE INSTITUTE
The Cedars-Sinai Regenerative Medicine Institute (RMI) brings together research faculty and clinicians to provide a true “bench to bedside” organization. We have five major programs of research: (i) Brain, (ii) Eye, (iii) Pancreas and Liver, (iv) Blood and (v) Skeletal. Working within each of these programs benefits from our core facilities with a focus on generating induced pluripotent stem cells from adult human skin samples and optimizing differentiation into various tissues of the human body of interest to the six programs. The pluripotent cells are used to both increase our understanding of human diseases through modeling, and provide a foundation for pre-clinical studies aimed at establishing and validating cellular therapeutic approaches to human illness.
Presenter: Clive Svendsen, Ph.D., Director, Cedars-Sinai Regenerative Medicine Institute
CELLULAR DYNAMICS INTERNATIONAL
Cellular Dynamics International, Inc. (CDI) is a leading developer of next-generation stem cell technologies for drug development, cell therapy, tissue engineering and organ regeneration. CDI harnesses its unique manufacturing technology to produce differentiated tissue cells from any individual’s stem cell line in industrial quality, quantity and purity. CDI is accelerating the adoption of pluripotent stem cell technology, adapting its methods to fit into standard clinical practice by the creation of individual stem cell lines from a standard blood draw. CDI was founded in 2004 by Dr. James Thomson, a pioneer in human pluripotent stem cell research at the University of Wisconsin-Madison.
Presenter: Robert Palay, Chairman and CEO, Cellular Dynamics International
Cytomedix, Inc. (OTCBB:CMXI) is a US-based company that is developing and commercializing autologous regenerative biotherapies that promote healing by harnessing the innate regenerative capacity of platelets and adult stem cells.
Presenter: Ed Field, COO, Cytomedix
Cytori Therapeutics is developing cell therapies based on autologous adipose-derived stem and regenerative cells (ADRCs) to treat cardiovascular disease and repair soft tissue defects. Preclinical data suggest ADRCs improve blood flow, moderate the immune response and keep tissue at risk of dying alive. Cytori’s cell therapies are made available by its proprietary Celution® System, which automates the extraction and preparation of clinical grade autologous ADRCs at the point-of-care. The Company has ongoing clinical trials for cardiovascular disease in the U.S. and Europe and is commercializing Celution® in Europe and Asia Pacific for soft tissue repair and translational research.
Presenter: Doug Arm, Senior Vice President, Operations, Cytori
DiscGenics™ is a development stage spinal therapeutics company using adult human disc-derived stem cells and tissue engineering techniques to treat patients debilitated by degenerative disc disease (DDD). Back pain is the second most common reason to visit one’s doctor, and costs an estimated $100 billion in diagnosis, management, and lost productivity each year. From our patented culture method comes the Discophere™, a therapeutic cluster of stem/progenitor cells that have been shown to differentiate and excrete the biological components needed to regenerate an intervertebral disc.
Presenter: Flagg Flanagan, CEO, DiscGenics
Eqalix has received exclusive commercial licensing rights from three prominent institutions for several groundbreaking technologies in Regenerative Medicine which have the potential to revolutionize the unmet needs in multiple commercial and therapeutic applications. These technologies introduce the second generation of products into development and address previously unmet medical needs in the Regenerative Medicine space. Using these technologies, Eqalix plans to develop and commercialize (a) plant-protein based nano-fiber scaffold for use in wound healing and aesthetic dermatology; (b) small-diameter hybrid vessels that foster the creation of a functional endothelium (c) 3-Dimensional tissue scaffolds with adjustable properties for organ and tissue replacement and repair.
Presenter: Joseph Connell, CEO, Eqalix
Healthpoint Biotherapeutics is focused on the development and commercialization of novel, cost-effective drugs and biologics for acute, chronic and burn-related wounds. Currently marketed products include the only approved biologics in wound care, Collagenase SANTYL® Ointment and REGRANEX® Gel, along with OASIS® Wound Matrix and OASIS® Ultra Tri-Layer Matrix, the industry’s leading extracellular matrix technology. R&D is focused on HP802-247, an investigational allogeneic living cell bioformulation. The Phase II-b trial, which was recently published in The Lancet, showed a >50% improvement in raw healing rate for treatment vs control at the end of the 12 week study in venous leg ulcer patients.
Presenter: Robert Bancroft, Executive Vice President, Strategic and Commercial Development, Healthpoint Biotherapeutics
Juventas Therapeutics is a privately held, clinical-stage biotechnology company developing factor-based regenerative therapies to treat life-threatening diseases. The company’s lead product, JVS-100 encodes Stromal cell-Derived Factor-1 (SDF-1) which has been shown to protect and repair tissue following ischemic injury by recruiting the body’s own stem cells to the damaged tissue, preventing cell death and promoting new blood vessel growth. Through activating natural stem cell based repair pathways within the patient, we eliminate the cost and complexity associated with current cellular therapies. Juventas is currently enrolling two Phase II clinical trials to test therapy efficacy in heart failure and critical limb ischemia patients.
Presenter: Rahul Aras, President and CEO, Juventas Therapeutics
MaxCyte is the leader in scalable and clinical cell transfection, bringing to market its patented flow electroporation technology. MaxCyte is applying its significant capabilities in the discovery, development, and manufacturing of virtually all classes of innovative therapeutics targeting a broad range of diseases. MaxCyte’s customers and partners utilize its technologies in the development and commercialization of cell-based therapies in regenerative medicine and active cell immunotherapies and in the discovery and development of protein drugs, monoclonal antibodies, vaccines, and small molecule drugs. This clinical-grade cell loading technology is fully developed, well validated and has received MF designation with CBER at U.S. FDA.
Presenter: Douglas Doerfler, President and CEO, MaxCyte
Medistem, Inc., is a clinical stage San Diego-based biotechnology company. We are focused on the development of our proprietary Endometrial Regenerative Cell (ERC) technology, which provides a universal donor adult stem cell platform. ERCs possess specialized abilities to stimulate new blood vessel growth and can differentiate into lung, liver, heart, brain, bone, cartilage, fat and pancreatic tissue. These unique properties have applications for treatment of Critical Limb Ischemia (CLI), congestive heart failure (CHF), neurodegenerative diseases, liver failure, kidney failure, and diabetes. ERCs have been cleared by the FDA to begin studies in the United States.
Presenter: Alan Lewis, CEO, Medistem, Inc.
Nanofiber Solutions, LLC is a medical device manufacturer of three-dimensional (3-D) scaffolds and cell culture products that advance biological sciences. Our platform technology provides synthetic structures that mimic physical structures found in the human body, specifically the extracellular matrix (ECM). By mimicking the ECM, our scaffolds increase cell acceptance and allow for the development of products that significantly aid basic research, improve commercial drug and therapeutic development, and serve as a platform for artificial implants. Our scaffolds currently support three major areas of medicine; tissue engineering, regenerative medicine and basic research.
Presenter: Ross Kayuha, CEO, Nanofiber Solutions
NeoStem, Inc. is an emerging technology leader in the fast-developing cell therapy market. Its business strategy combines a state-of-the-art contract development and manufacturing subsidiary, Progenitor Cell Therapy, LLC, with a medically important cell therapy product development program. NeoStem’s most clinically advanced therapeutic, AMR-001, is being developed at its Amorcyte subsidiary, which is enrolling patients in a Phase 2 trial for preservation of heart function after a heart attack. Athelos Corporation, also a NeoStem subsidiary, is in early stage clinical exploration of a T-cell therapy for autoimmune conditions. Pre-clinical assets include our VSELTM Technology platform and our mesenchymal stem cell product candidate for regenerative medicine.
Presenter: Robin Smith, M.D., M.B.A., Chairman and CEO, NeoStem, Inc.
NEW YORK STEM CELL FOUNDATION
NYSCF is combining the depth of a highly focused research laboratory with the breadth of a wide-ranging philanthropic organization and has profoundly affected the entire field of stem cell research. Since its establishment, NYSCF has conducted the most advanced stem cell research in the U.S. in its laboratory and in collaboration with major medical research institutions. Co-founded in 2005 by Susan L. Solomon, NYSCF has raised nearly $100 million for stem cell research worldwide, primarily through private philanthropy. By creating its own state-of-the-art laboratory and by supporting and pursuing only the most advanced stem cell research, NYSCF has catalyzed this promising field.
Presenter: Susan Solomon, Co-founder and CEO, New York Stem Cell Foundation
Organovo (OTCQB: ONVO) is a three-dimensional (3D) biology company focused on delivering breakthrough bioprinting technology and creating tissue on demand for research and medical applications. The company’s NovoGen 3D bioprinting technology is a platform that works across all tissue and cell types. Organovo is helping pharmaceutical partners develop human biological disease models in 3D that enable drug discovery and development. Organovo’s bioprinting technology can also be used to create surgical tissues as direct therapy. Organovo leads the way in solving complex medical research problems and building the future of medicine.
Presenter: Eric David, Chief Strategy Officer, Organovo
Owl biomedical is an emerging company commercializing an innovative high-speed, disposable cartridge-based cell sorting platform called the Nanosorter®. The Nanosorter® enables rapid processing of large numbers of cells safely without aerosols and in a simple, readily deployable and easy to use system. The Nanosorter® platform uniquely combines the world’s fastest microvalve (fabricated within a microchip) and the well-proven principles of fluorescence-activated sorting of cells. Cells purified using the Nanosorter® can be employed for a wide variety of commercial applications, including: adoptive immunotherapy for treatment of cancer, stem cell therapies for regenerative medicine (e.g. stroke, limb ischemia, wound healing), cell-based cancer diagnostics, and a wide variety of cell-based applied research applications. Owl biomedical was founded in 2011 and is based in Santa Barbara, California.
Presenter: Jim Linton, Ph.D., M.B.A., President and Chief Business Officer, Owl Biomedical
Q Therapeutics is harnessing the power of stem cells to treat debilitating diseases of the central nervous system, which encompass a broad range of diseases including ALS, multiple sclerosis, spinal cord injury, stroke, cerebral palsy, Parkinson’s and Alzheimer’s diseases. Q-Cells® function as ‘mini-factories’ to repair neurons, utilizing multiple pathways including myelination, growth factor production and toxin removal. By utilizing the natural support and repair mechanisms of healthy CNS, safety and efficacy are enhanced. Q has a strong proprietary position with 16 patents issued and 12 pending. Our products address a multi-billion dollar market opportunity for which traditional drugs have failed.
Presenter: Deborah Eppstein, Ph.D., President and CEO, Q Therapeutics
The Regen BioPharma business model is to take multiple stem cell therapeutics to and through the human “safety and signal of efficacy” stage (Phase I/II clinical trials). The approach is a highly focused analysis of issued patents in regenerative medicine, identification and acquisition of undervalued assets that have demonstrated proof of concept, and forming companies around these assets. Having assembled a core infrastructure specialized in obtaining regulatory approval and executing clinical trials in cell therapy, we aim to act as a “superincubator” that within 18 – 24 months grows technologies from laboratory to an asset ready for spin-off or sale to feed the pipeline of Big Pharma.
Presenter: J. Christopher Mizer, President, Regen BioPharma, Inc.
REGENERATIVE PATCH TECHNOLOGIES
Regenerative Patch Technologies was formed to advance development of a composite RPE cell-membrane technology currently supported by a disease team award to the University of Southern California, the University of California, Santa Barbara, and the California Institute of Technology from the California Institute of Regenerative Medicine. The technology utilizes polarized RPE cells derived from human embryonic stem cells together with an ultrathin biocompatible parylene membrane as a replacement for the defective Bruch’s membrane RPE cell complex often observed in patients with the atrophic form of age related macular degeneration. The project is progressing through preclinical studies to enable IND filing in 2014.
Presenter: Jane Lebkowski, President and CSO, Regenerative Patch Technologies
RhinoCyte™, Inc., is a biopharmaceutical company focused on developing innovative stem cell therapies for neurodegenerative diseases. Founded in 2005, the company has raised $5 million to date to develop this transformational technology to treat Spinal Cord Injury and Parkinson’s Disease to extend and enhance the quality of human life. The first RhinoCyte™ product is for Spinal Cord Injury (SCI). The IND submission for SCI is 4Q12. RhinoCyte™ is currently raising a $10 Million Series B financing to fund the Spinal Cord Injury Phase I Clinical Trial program and facilitate the clinical status of the Parkinson’s disease product.
Presenter: Teresa Leezer, COO, RhinoCyte
Sangamo is a clinical stage biopharmaceutical company focused on the development and commercialization of engineered zinc finger DNA-binding proteins to build a new class of therapeutic products that function at the DNA level. Our mission is to develop ZFP Therapeutics® for rare and monogenic diseases and other diseases with high unmet medical needs where our product has a differential technical advantage while providing positive outcomes for patients not achievable by current treatment paradigms. In February 2012, we entered into a strategic collaboration with Shire plc to develop and commercialize ZFP Therapeutics® for the treatment of hemophilia and other monogenic diseases.
Presenter: Edward Lanphier, President and CEO, Sangamo BioSciences
Sistemic’s world leading expertise is on applying microRNA profiling in context with the biology, to areas where there is currently an unmet need for sophisticated tools delivering instructive and robust knowledge of the cell system. From their SistemQC™ platform they have derived an extensive suite of tools for the cell therapy, gene therapy and bioprocessing community. SistemQC™, molecularly characterises cells including stem cells as well as aids in the optimization and monitoring (QC) of the manufacture process. More specifically the initial focus of SistemQC™ by clients has been on generation of microRNA based ID markers, purity & potency assessment and manufacture monitoring & optimization.
Presenter: Jim Reid, Chairman and CEO, Sistemic
STANFORD CARDIOVASCULAR INSTITUTE
Stanford Cardiovascular Institute (CVI) is the nucleus for cardiovascular research at Stanford University. Formed in 2004, the Cardiovascular Institute is home to Stanford’s myriad cardiovascular-related adult and pediatric research, clinical, and educational programs, centers and laboratories, as well as over 500 Stanford basic scientists, graduate students, clinician scientists, and other researchers in heart and vessel disease and prevention. Within the area of cardiac regenerative medicine, the Stanford CVI has significant research endeavors involving human pluripotent stem cells for (1) cardiovascular disease modeling, (2) drug screening and discovery, and (3) personalized cell therapy. Recently, Stanford CVI investigators received a $20 million CIRM Disease Team Therapy Award for performing pre-IND work that would enable the first-in-man clinical trial involving injection of human embryonic stem cell-derived cardiomyocytes for patients with heart failure.
Presenter: Joseph Wu, Associate Professor, Cardiovascular Medicine, Stanford University School of Medicine
StemBioSys is a development-stage company with novel technologies to isolate and expand specialized stem cell (SC) populations, including large numbers of elusive, pluri-potent, embryonic-like stem cells (ELSCs) from cord blood. The core technology platform is a patented ECM, produced by bone marrow MSCs, which replicates their native microenvironment, promotes proliferation of various SC beyond current methods and preserves SC phenotypes (“stemness”). StemBioSys ELSCs have generated tissues in vivo from all three germ layers, providing a viable research and clinical alternative to both hESCs and iPS cells. These technologies help overcome major obstacles to developing autologous and allogenic stem cell therapies.
Presenter: Sy Griffey, Ph.D., Chief Operating Officer, StemBioSys
StemCells, Inc. is engaged in the research, development, and commercialization of cell-based therapeutics and tools for use in stem cell-based research and drug discovery. StemCells’ lead therapeutic product candidate, HuCNS-SC® cells (purified human neural stem cells), is currently in clinical development for a broad range of central nervous system disorders, including myelination disorders, spinal cord injury, and age-related macular degeneration. The Company has completed two clinical studies, is currently conducting two other trials, and is pursuing preclinical studies in Alzheimer’s disease. StemCells also markets stem cell research products under the SC Proven® brand.
Presenter: Martin McGlynn, President and CEO, StemCells, Inc.
TAP Biosystems provides advanced automated systems for cell therapy scale-up and manufacture. At SCMOM 2012 TAP will be showcasing a range of systems including the CellBase CT™ automated cell culture system for ex vivo expansion of autologous cell therapy products. Plus the Cellmate™ system for batch production of cells in rollers bottles and the RAFT™ system for producing biomimetic 3D tissue models in standard plate formats. Staff from TAP will also be available to discuss our collaborative working process to help businesses create new cell therapy scale-up and GMP manufacturing systems.
Presenter: Rosemary Drake, Chief Scientific Officer, TAP Biosystems
TheraBiologics is a privately-held, clinical-stage biopharmaceutical company founded in 2011 to harness the natural properties of human neural stem cells (NSCs) to migrate to and penetrate tumors. The Company’s TBX-NSC™ platform enables the targeted delivery of potent therapeutics to tumors. TheraBiologics Phase I lead program, TBX-01, is the first–in-human clinical trial of an FDA-approved, genetically modified NSC line for cancer treatment. TheraBiologics second lead product candidate,TBX-02, is undergoing preclinical testing and funded by an $18 million CIRM award to Dr. Aboody, Associate Professor, City of Hope, and TheraBiologics founder.
Presenter: Karen Aboody, M.D., Founder and CSO, TheraBiologics
TiGenix NV (NYSE Euronext: TIG) is a leading European cell therapy company with a commercial product and an advanced clinical stage pipeline of adult stem cell programs. The company’s lead product, ChondroCelect®, for cartilage repair in the knee, is the first and only approved cell-based product in Europe. TiGenix’s stem cell programs are based on a validated platform of allogeneic expanded adipose-derived stem cells (eASCs) targeting autoimmune and inflammatory diseases. The company has initiated a Phase III clinical trial in complex perianal fistulas in patients with Crohn’s disease, is conducting a Phase IIa trial in rheumatoid arthritis, and successfully concluded a Phase I trial to investigate the potential of intra-lymphatic administration of eASCs for autoimmune disorders.
Presenter: Eduardo Bravo, CEO, TiGenix
UC DAVIS INSTITUTE FOR REGENERATIVE CURES
UC Davis is playing a leading role in regenerative medicine, with nearly 150 scientists working on a variety of stem cell-related research projects at campus locations in both Davis and Sacramento. The UC Davis Institute for Regenerative Cures, a facility supported by the California Institute for Regenerative Medicine (CIRM), opened in 2010 on the Sacramento campus. This $62 million facility is the university’s hub for stem cell science. It includes Northern California’s largest academic Good Manufacturing Practice laboratory, with state-of-the-art equipment and manufacturing rooms for cellular and gene therapies. UC Davis also has a Translational Human Embryonic Stem Cell Shared Research Facility in Davis and a collaborative partnership with the Institute for Pediatric Regenerative Medicine at Shriners Hospital for Children Northern California. All of the programs and facilities complement the university’s Clinical and Translational Science Center, and focus on turning stem cells into cures.
Presenter: John Laird, M.D., Professor, Medicine and Medical Director, UC Davis Vascular Center, UC Davis Health System
Presenter: Vicki L. Wheelock, M.D., Clinical Professor, Neurology, Director, HDSA Center of Excellence, Principle Investigator, CIRM Grant DR2A-05415, UC Davis Health System
ViaCyte, Inc., a leader in the emerging field of regenerative medicine, is headquartered in San Diego, California. ViaCyte’s innovative product is based on the differentiation of stem cells into pancreatic beta cell precursors (PEC-01), with subcutaneous implantation in a retrievable and immune-isolating encapsulation medical device. Once implanted, the precursor cells mature into endocrine cells that secrete insulin in a regulated manner to control blood glucose levels. ViaCyte’s goal is a product that can free Type 1 and 2 diabetic patients from long-term insulin dependence. ViaCyte has received substantial financial support from both CIRM and JDRF.
Presenter: Paul Laikind, President and CEO, ViaCyte