Company Presentations


40+ Presenting Companies Conducting 100+ Clinical Trials with 25+ Products Already on the Market!

The 4th Annual Partnering Forum at Stem Cell Meeting on the Mesa includes a pipeline loaded with partnering opportunities. Collectively, the initial slate of 40+ presenting companies are conducting 100+ clinical trials with an additional 135 studies currently in the pre-clinical phase – not to mention their 25+ products already on the market! Altogether these companies are working on 150+ unique products with an extensive range of possible therapeutic applications. With 10+ hours of presentations over the course of two days, this meeting provides attendees a unique opportunity to forge collaborations with regenerative medicine’s top industry leaders.

BioLife-Solutionsblue bird bio

2014 Company Presenters


Dominick Colangelo, President & CEO
Ann Arbor, MI
Founded in 1989, Aastrom Biosciences is dedicated to the development of stem cell treatments for critical cardiovascular diseases. Aastrom is currently evaluating its autologous cellular therapies in U.S. clinical trials in the treatment of dilated cardiomyopathy (DCM). These critical diseases are associated with significant morbidity and mortality and very limited treatment options.


Dan Menichella, Chief Business Officer
Alachua, FL
AGTC is developing cures for rare lung and eye diseases, offering hope to patients with unmet medical needs. With a highly specialized team of physicians and researchers, the Company is use cutting-edge techniques to develop treatments for patients that have diseases caused by broken genes. AGTC uses AAV gene therapy, which replaces those broken genes with normal functional genes, allowing a patient’s own body to produce proteins to treat their illness. A single injection provides long-lasting treatment, leading to a better quality of life for patients worldwide.


Gil Van Bokkelen, Ph.D., Chairman & CEO
Cleveland, OH
Athersys is a clinical-stage biotechnology company developing novel and proprietary best-in-class therapies designed to extend and enhance the quality of human life. The Company’s focus is on the treatment of medical conditions where there is significant unmet clinical need. Athersys is developing MultiStem®, a patented, adult-derived “off-the-shelf” stem cell product platform for multiple disease indications in the areas of inflammatory and immune, neurological and cardiovascular disease. The Company has two ongoing clinical studies – a Phase II study in ulcerative colitis being conducted with Pfizer and a Phase II study in ischemic stroke – and overall five clinical-stage programs in inflammatory bowel disease, stroke, graft-versus-host disease, acute myocardial infarction and solid organ transplant. Athersys has forged a network of strategic alliances and collaborations with leading pharmaceutical and biotechnology companies, as well as world-renowned research and clinical institutions, in the U.S. and Europe to further develop its platform and products.


Hans Hull, SVP, Legal & Corporate Development
Menlo Park, CA
Avalanche is a clinical-stage biotechnology company focused on discovering and developing novel gene therapies to transform the lives of patients with sight-threatening ophthalmic diseases. Using a next generation gene therapy platform, the Ocular BioFactory™, the Company is developing products designed to provide long-term benefit or a functional cure by inducing a sustained expression of a therapeutic protein with a one-time administration in the eye.


Mike Rice, CEO
Bothell, WA
BioLife Solutions develops, manufactures and markets hypothermic storage and cryopreservation solutions and precision thermal shipping products for cells, tissues and organs. The Company’s proprietary HypoThermosol® and CryoStor® platform of solutions are highly valued in the regenerative medicine, biobanking and drug discovery markets. BioLife’s biopreservation media products are serum-free and protein-free, fully defined and are formulated to reduce preservation-induced cell damage and death. The Company’s enabling technology provides commercial companies and clinical researchers significant improvement in shelf life and post-preservation viability and function of cells, tissues and organs. BioLife also performs contract aseptic media formulation, fill and finish services. To date, the Company’s proprietary biopreservation media products have been incorporated into over 100 hospital-approved and clinical trial stage regenerative medicine products and therapies.


Jeffrey Walsh, COO
Cambridge, MA
bluebird bio is a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases. bluebird bio has two clinical-stage programs in development. The most advanced product candidate, Lenti-D, is in a recently-initiated Phase II/III study, the Starbeam Study, for the treatment of childhood cerebral adrenoleukodystrophy (CCALD), a rare, hereditary neurological disorder affecting young boys. The next most advanced product candidate, LentiGlobin, is currently in two Phase I/II studies, one in the U.S. (the Northstar Study) and one in France (HGB-205), for the treatment of beta-thalassemia major. The Phase I/II HGB-205 study also allows enrollment of patients with sickle cell disease, and bluebird bio is planning a separate U.S. sickle cell disease trial (HGB-206).


Linda Marbán, Ph.D., CEO
Beverly Hills, CA
Los Angeles-based Capricor Therapeutics is a clinical-stage biotechnology company focused on the development and commercialization of regenerative medicine and large molecule products for the treatment of diseases. Capricor’s lead product candidate, an allogeneic cardiosphere-derived cell (CDC) product, CAP-1002, aims to attenuate and potentially improve damage to the heart caused by a heart attack and is currently under evaluation in the Phase I/II ALLSTAR clinical trial of patients 30-days to 1-year after a heart attack. Additionally, Capricor plans to explore development of exosome technology as a next generation regenerative medicine platform in a variety of cardiovascular and non-cardiovascular areas. Groundbreaking preclinical research has demonstrated that exosomes extracted from Capricor’s CDCs reduced scar tissue caused by a heart attack and prompted myocardial regeneration in preclinical models of ischemic heart disease.


Christian Homsy, CEO
Mont-Saint-Guibert, Belgium
(CARD:EN Brussels)
Cardio3 BioSciences is a leading biotechnology company focused on the discovery and development of regenerative and protective therapies for the treatment of cardiac diseases. The Company’s lead product candidate, C-Cure®, is a highly innovative stem cell approach for the treatment of heart failure, one of the world’s most pressing unmet medical needs. Generated through a proprietary technological platform, the C3BS-CQR-1 programme is designed to direct the patient’s own stem cells into new heart cells with the potential to rebuild the heart. Cardio3 BioSciences has also developed C-Cath®ez, the most technologically advanced injection catheter with superior efficiency of delivery of bio therapeutic agents into the myocardium. Cardio3 BioSciences was founded in July 2007 and is based in Mont-Saint-Guibert in the Walloon region of Belgium. The Company leverages research collaborations in the U.S. and in Europe with Mayo Clinic.


Clive Svendsen, Ph.D., Director & Professor, Board of Governors Regenerative Medicine Institute
Los Angeles, CA
(Non-Profit Hospital)
Regenerative medicine is a new and developing field that aims to restore function in diseased or aged tissues through either revitalizing existing cells, or the transplantation of new cells-stem cells. These cells are of great interest to regenerative medicine. They lie deep within most tissues of the adult body, but are often difficult to manipulate or expand outside the body. Induced pluripotent stem (iPS) cells are stem cells from the adult human body that have been reprogrammed back to an embryonic stem cell state. These are important for regenerative medicine as they can be derived from a single patient, turned into any tissue of the body and then used as a source of autologous cells for repair – thus avoiding immune rejection issues and may be used as novel and important models of human disorders. The use of iPS cells is not burdened with the ethical issues associated with human embryos.


Krisztina Zsebo, Ph.D., CEO
San Diego, CA
Celladon is a clinical-stage biotechnology company developing novel therapies for diseases with tremendous unmet medical needs. The Company’s lead gene therapy product candidate MYDICAR® targets calcium dysregulation in cardiomyocytes by increasing SERCA2a enzyme levels in advanced heart failure. In a placebo controlled Phase IIa trial, MYDICAR® demonstrated an 88% reduction of hospitalizations for heart failure and a trend in improved overall survival. Benefit in clinical outcomes was also supported by improvement in patients’ heart failure symptoms, exercise tolerance, serum biomarkers and cardiac function. MYDICAR® recently received Breakthrough Therapy designation by the FDA, and the EMA indicated that if MYDICAR® demonstrates a substantial and highly significant treatment effect, and no untoward effects attributable to MYDICAR® are observed, a safety database of approximately 205-230 MYDICAR® treated subjects may be sufficient to allow for acceptance of an MAA. MYDICAR® is currently in a 250-patient Phase IIb trial with results expected in April 2015.


Robert Palay, CEO
Madison, WI
Cellular Dynamics International is a leading developer of fully functional human cells derived from induced pluripotent stem (iPS) cells. The Company’s iCell® and MyCell® product lines provide industrial quantities of high quality, highly pure human cells enabling disease modeling, drug discovery, toxicity testing and regenerative medicine research.


Matthew Plavan, CEO
Rancho Cordova, CA
Cesca Therapeutics is engaged in the research, development and commercialization of autologous cell-based therapeutics for use in regenerative medicine. The Company is a leader in developing and manufacturing automated blood and bone marrow processing systems that enable the separation, processing and preservation of cell and tissue therapy products.


Koji Kuchiishi, Chairman, President & CEO
Tokyo, Japan
Founded in 2010, Cyfuse Biomedical K.K. is a Japan-based start-up focused on scaffold-free 3D tissue engineering. Cyfuse successfully developed the bio 3D printer Regenova, a state-of-the-art robotic system that enables fully automated fabrication of three-dimensional artificial tissues from living cells. The Company’s goal is to facilitate innovative therapeutic approaches through new bioengineering technologies to make regenerative medicine a reality for patients around the world.


Martin Rosendale, CEO
Gaithersburg, MD
Cytomedix is an autologous regenerative therapies company commercializing innovative platelet technologies for wound care. The Company markets the AutoloGel™ System, a device for the production of autologous platelet rich plasma (PRP) gel for use on a variety of exuding wounds.


Marc Hedrick, M.D., President & CEO
San Diego, CA
Cytori Therapeutics is a cell therapy company dedicated primarily to the development of novel treatments for cardiovascular disease and soft tissue injuries and burns. The Company has a global product development strategy focused on the U.S. cardiovascular disease market. In the U.S. Cytori’s goal is to bring cell therapy to market for treatment of chronic ischemic heart failure through Cytori-sponsored clinical development efforts and to develop a treatment for thermal burns combined with radiation injury under a contract from BARDA, a division of the U.S. Department of Health and Human Services.


Flagg Flanagan, Chairman & CEO
Salt Lake City, UT
DiscGenics is a biotechnology company developing advanced spinal therapeutics to treat patients with diseases of the intervertebral disc. DiscGenics is the only company to utilize therapeutic progenitor cells derived from disc to treat the disc. The Company’s allogeneic methodology allows DiscGenics to derive many doses from each adult tissue donation. The Company’s first product, Injectable Discogenic Cell Therapy (IDCT), is in preclinical development for the treatment of moderate degenerative disc disease. This cellular treatment offers a cost-effective and non-surgical solution to patients who currently have few treatment options. Further, the treatment may delay the progression towards costly and often ineffective surgical interventions necessary at the late stages of degeneration. A follow-on product utilizing a different scaffold carrier will be for post-discectomy patients and is currently being researched.


David Pernock, Chairman & CEO
Exton, PA
Fibrocell Science is an autologous cell therapy company primarily focused on developing first-in-class treatments for rare and serious skin and connective tissue diseases with high unmet medical needs. Based on its proprietary autologous fibroblast technology, Fibrocell is pursuing breakthrough medical applications of azficel-T for restrictive burn scarring and vocal cord scarring. The company’s collaboration with Intrexon Corporation, a leader in synthetic biology, includes using genetically-modified fibroblasts for treating rare and serious skin and connective tissue diseases for which there are currently no approved products.


David Green, CEO
Hilliston, MA
Harvard Apparatus Regenerative Technology (HART) is a clinical-stage biotechnology company making regenerated organs for transplant. The Company’s first product, the HART-Trachea, is intended to be used to restore the structure and/or function of a severely damaged trachea. The HART-Trachea is comprised of autologous bone marrow cells seeded on the Company’s proprietary InBreath synthetic scaffold in its proprietary InBreath organ bioreactor. To date, HART’s InBreath bioreactor technology has been used to treat eight human patients, and the five most recent of these transplants also used HART’s InBreath scaffold. The Company believes that its HART-Trachea could enable surgeons to address nearly all life-threatening constrictions of the trachea, and expects to submit its IND application for HART-Trachea to the FDA by the end of 2015. In addition to its trachea product, HART is also working with major medical research institutions to regenerate the lungs, esophagus, lower GI, heart valves and heart.


Gail Naughton, Ph.D., Chairman & CEO
San Diego, CA
Histogen is a regenerative medicine company manufacturing and developing innovative therapies based on the products of cells grown under simulated embryonic conditions. Through Histogen’s proprietary technology process, including conditions of low oxygen and suspension, newborn cells are encouraged to naturally produce vital proteins and growth factors which are characteristic of young, rapidly developing tissue and which can stimulate stem cells in the patient’s own body to regenerate into new tissue. Using this patented tissue-engineering platform, Histogen has developed a rich pipeline of unique commercialization opportunities for both aesthetic and therapeutic applications. The Company’s lead product, Hair Stimulating Complex (HSC) has shown success in two Company-sponsored clinical trials as an injectable treatment for alopecia. In addition, Histogen’s human multipotent cell conditioned media can be found in skincare products including ReGenica, which is distributed by Suneva Medical in partnership with Obagi Medical Products.


Tom Ulich, M.D., Chief Scientific Officer
Cambridge, MA
InVivo Therapeutics is a pioneering biomaterials and biotechnology company with a focus on treatment of spinal cord injuries. The Company was founded in 2005 with proprietary technology co-invented by Robert Langer, ScD., Professor at Massachusetts Institute of Technology (MIT), and Joseph Vacanti, M.D., who is affiliated with Massachusetts General Hospital. InVivo is developing biocompatible neuro-spinal scaffolds to treat acute SCI and neuro-spinal scaffolds plus stem cells to treat chronic SCI. Data on InVivo’s scaffold and scaffold plus stem cells technology was first published in the Proceedings of the National Academy of Sciences (PNAS) in 2002 and has since been demonstrated to successfully promote functional recovery following traumatic spinal cord injury in several models. Data from InVivo’s pilot non-human primate study was published in the Journal of Neuroscience Methods in 2010, which earned the David S. Apple Award from the American Spinal Injury Association for its outstanding contribution to spinal cord injury medicine.


Mitchell Seyedin, Ph.D., Executive Chairman
St. Louis, MO
A leading regenerative medicine company focused on developing novel orthobiologic products, the Company has two late-stage development programs targeting areas of significant unmet medical need in spine and sport medicine. The lead product candidates are NuQu for the treatment of discogenic back pain and RevaFlex for articular knee cartilage repair and restoration. ISTO’s cartilage regeneration platform uses juvenile cartilage cells that are expanded using a proprietary manufacturing process. Juvenile cartilage cells possess vastly superior regeneration potential compared to adult cartilage cells. In addition to the cartilage regeneration platform, ISTO has developed and markets a synthetic biomaterial for bone generation which is primarily used in spinal fusion applications. InQu is FDA 510(k) cleared for use as a bone graft extender for fusion in all areas of the spine. InQu has been used in over 30,000 procedures and has demonstrated an excellent safety profile.


Henry Klassen, M.D., Ph.D., Founder
Newport Beach, CA
jCyte is developing a progenitor cell-based product for use in retinal degenerative diseases, specifically blinding conditions involving the loss of photoreceptors. The initial target disease is the orphan disease known as retinitis pigmentosa (RP) and the follow-on disease target is age-related macular degeneration (AMD). jCyte’s novel translational work has received substantial support from the California Institute of Regenerative Medicine (CIRM), first as an Early Translational Grant in the amount of $4 million in 2010 and then in the form of a Disease Team Therapy Development Award for an additional $17.1 million in 2012. This funding provides for IND-enabling preclinical work as well as early-stage clinical trials. jCyte is one of a small number of entities pioneering the application of regenerative medicine-based technologies to unmet medical need in the retina. The immediate goal is to obtain regulatory approval from the FDA for clinical testing in retinitis pigmentosa.


Marc Penn, M.D., Ph.D., Founder & Chief Medical Officer
Cleveland, OH
Juventas Therapeutics is a private clinical-stage company developing novel therapies for ischemic cardiovascular disease. Founded in 2007 with an exclusive license from Cleveland Clinic, Juventas has transitioned its therapeutic platform from concept into Phase II clinical trials for heart failure and critical limb ischemia. The Company’s lead product, JVS-100, is a non-viral DNA plasmid that encodes for stromal cell-derived factor-1 (SDF-1). SDF-1 has been shown to significantly increase end-organ function following tissue injury by promoting cell survival, recruiting endogenous stem cells to the damaged region and promoting new blood vessel growth. The SDF-1 repair pathway is well conserved throughout end-organ systems providing the opportunity to impact a broad range of diseases. Target cardiovascular clinical indications address large markets with high unmet medical need and significant market potential.


Margo Roberts, Ph.D., Chief Scientific Officer
Santa Monica, CA
Kite Pharma is developing a pipeline of product candidates for the treatment of advanced solid and hematological malignancies using the Company’s therapeutic platform – engineered Autologous Cell Therapy (eACT™) – in which a patient’s own T cells, or white blood cells, are engineered to recognize and destroy their cancer. The patient’s immune system, particularly T cells, plays a critical role in identifying and killing cancer cells. Kite Pharma’s eACT™ technology involves the genetic engineering of T cells to express either chimeric antigen receptors (CARs), or T cell receptors (TCRs). The Company’s technology has been developed in part through a collaboration with the National Cancer Institute, Surgery Branch through a cooperative research and development agreement (CRADA). Kite Pharma is advancing a pipeline of proprietary eACT™-based product candidates to create personalized therapies targeting a broad range of indications in cancer.


Doug Doerfler, President & CEO
Gaithersburg, MD
MaxCyte specializes in cell modification technologies to enable the discovery, development, manufacturing and delivery of innovative cell-based therapeutic products. MaxCyte is pioneering the use of mRNA for Chimeric Antigen Receptor (CAR) immunotherapies for solid cancers with Drs. Carl June and Dario Campagna. Early in 2013, MaxCyte received U.S. patent 8,450,112 providing broad coverage for transiently modifying unexpanded cells (specifically, unstimulated resting PBMCs) to express CARs for treating cancer. This approach, building on the work with Dr. June and others and pioneered by MaxCyte, enables the dramatic reduction of costs and processing times (from two weeks to less than one day), leveraging existing leukopheresis infrastructure to create a rapid, truly commercializable and cost-effective form in mRNA CAR cancer therapies.


Antonio Lee, Ph.D., CEO & Managing Director
Rockville, MD
(078160: KOSDAQ)
MEDIPOST is a leading stem cell biotechnology company focused on developing stem cell therapeutics using human Umbilical Cord Blood-derived Mesenchymal Stem Cells (hUCB-MSCs) to meet unmet medical needs such as those related to regenerative or functional recovery of knee articular cartilage, the nervous system, the pulmonary system and hematopoietic transplantation engraftment areas. CARTISTEM® is the world’s first allogeneic stem cell product with a Biologics License Application (BLA) approved by the Ministry of Food and Drug Safety (MFDS) Korea (former KFDA) for the regeneration of knee cartilage for the sufferers of degenerative osteoarthritis. CARTISTEM® contains multi-potent human Umbilical Cord Blood-derived Mesenchymal Stem Cells (hUCB-MSCs) with unique characteristics including not provoking an immune-response when transplanted into unrelated recipients. This allows large-scale manufacturing of CARTISTEM® using freshly isolated MSCs from the voluntarily donated cord blood units.


Donna Skerrett, M.D., Chief Medical Officer
Melbourne, Australia
Mesoblast Limited is developing novel biotherapeutics derived from its proprietary adult stem cell-based technologies. The Company’s technology platforms have the potential to deliver a diverse portfolio of clinical-stage products to treat a broad range of conditions with major unmet medical needs. Its strategic product development focus is in four major and distinct areas – systemic inflammatory conditions, cardiovascular diseases, orthopedic diseases of the spine and oncology conditions. Mesoblast’s corporate strategy is to: leverage proprietary cell-based and complementary biologic technologies to develop products for unmet medical needs; bring multiple products to market within a parallel timeframe; underpin future financial growth through investing in manufacturing operations; and enhance the likelihood of commercial success through strategic partnerships. Mesoblast’s strong cash reserves ($242 million at 31 March 2014) enable simultaneous product development with future revenues expected from milestone payments, distribution arrangements and direct sales.


Thomas Koob, Ph.D., Chief Scientific Officer
Marietta, GA
MiMedx®, the premier processor of regenerative biomaterial products and implants from human amniotic membrane, has distributed hundreds of thousands of amniotic tissue grafts worldwide. Profound clinical outcomes have been achieved in therapeutic areas including ophthalmology, spine, wounds, dental, orthopaedic, surgery, sports medicine and urology. With this innovative allograft that promotes bioactive healing, MiMedx® believes its intellectual property and proprietary processing technique strategically positions the Company as the leader in this area of regenerative medicine.


Robin Smith, M.D., Chairman & CEO
New York, NY
NeoStem is a leader in the emerging cellular therapy industry, pursuing the preservation and enhancement of human health globally through the development of cell-based therapeutics that prevent, treat or cure disease by repairing and replacing damaged or aged tissue, cells and organs and restoring their normal function. The business includes the development of novel proprietary cell therapy products as well as a revenue-generating contract development and manufacturing service business. This combination has created an organization with unique capabilities for cost effective in-house product development and immediate revenue and cash flow generation.


Sharon Presnell, Ph.D., Chief Technology Officer
San Diego, CA
Organovo designs functional human tissues. The Company’s bioprinting technology enables the creation of 3D tissues that more accurately reproduce native tissues. Organovo’s focus is on developing a range of tissue and disease models for medical research and therapeutic applications.


Lode Debrabandere, Ph.D., CEO
Columbia, MD
Through over 20 years of research, experience and understanding, Osiris Therapeutics has led the way in mesenchymal stem cell (MSC) science and the clinical application of cellular therapies. From commercializing its first generation implantable product, Osteocel, to achieving the Company’s initial goal, when founded in 1992, of developing the world’s first approved stem cell drug, remestemcel-L for graft versus host disease, Osiris has written the book on regenerative medicine. Today, as the leading stem cell company, Osiris continues to address unmet medical needs with innovative approaches in developing and marketing products in wound care, orthopedics and sports medicine. Never forgetting that the Company’s products are differentiated by scientific excellence, Osiris’ current product line includes Grafix® for acute and chronic wounds, Cartiform®, a viable cartilage mesh for cartilage repair, and OvationOS®, a viable bone matrix used for bone repair and regeneration.


Kyriacos Mitrophanous, Ph.D., Head of Research
Oxford, United Kingdom
Oxford BioMedica is a biopharmaceutical company developing innovative gene-based medicines and therapeutic vaccines that aim to improve the lives of patients with high unmet medical needs. The Company’s technology platform includes a highly efficient gene delivery system (LentiVector®), which has specific advantages for targeting diseases of the central nervous system and the eye, and a unique tumor antigen (5T4), which is an ideal target for anti-cancer therapy.


Eric Halioua, CEO
Mont-Saint-Guibert, Belgium
Promethera Biosciences is a pharmaceutical company that develops innovative cell therapies for the treatment of liver disease. Promethera® HepaStem is a cell therapy product based on the use of allogeneic stem cells isolated from healthy adult human livers, Heterologous Human Adult Liver Progenitor Cells (HHALPC). These cells can be used to treat a wide variety of liver diseases, from rare inborn metabolic diseases (which can be classified as orphan diseases and mainly affect children), to acquired deficiencies affecting adults, such as liver fibrosis. This treatment has already received the orphan drug designation from the European Medicines Agency and the FDA for the treatment of two very debilitating pediatric pathologies. Promethera Biosciences is conducting a prospective, open label, multicenter, partially randomized Phase I/II study testing one cycle of Promethera® HepaStem designed to evaluate the safety and preliminary efficacy in Urea Cycle Disorders and Crigler-Najjar Syndrome patients.


John Martin, Executive Chairman
Gordon, Australia
Regeneus is a Sydney-based regenerative medicine company founded in August 2007 that is focused on using the regenerative capacities of adipose-derived cells to develop innovative cell therapies for humans and animals. Regenerative medicine is a rapidly growing multidisciplinary specialty that is focused on the repair or regeneration of cells, tissues and organs. The primary goal is to enhance the body’s natural ability to replace tissue damaged or destroyed by injury or disease.


Michael Hunt, CEO
Guildford, England
ReNeuron is a leading clinical-stage cell therapy development business. Based in the U.K., its primary objective is the development of novel cell-based therapies targeting areas of significant unmet or poorly met medical need. ReNeuron has used its unique stem cell technologies to develop cell-based therapies for significant disease conditions where the cells can be readily administered “off-the-shelf” to any eligible patient without the need for additional immunosuppressive drug treatments. The Company’s therapeutic candidates for stroke disability and critical limb ischemia are in clinical development and its cell-based treatment for blindness-causing diseases of the retina is currently in pre-clinical development. ReNeuron is also advancing a proprietary platform technology to exploit nanoparticles (exosomes) secreted by stem cells as potential new drug candidates targeting indications in tissue repair, fibrosis and cancer.


David Hall, CEO
Vancouver, BC
RepliCel is a regenerative medicine company focused on autologous cell therapies that address diseases caused by a deficit of healthy cells required for healing and function such as chronic tendinosis, damaged skin and pattern baldness. The Company’s RCT-01 and RCS-01 therapies treat chronic tendinosis and damaged skin using fibroblasts isolated from the sheath of the hair follicle. RCH-01 is another hair follicle derived cell therapy for the treatment of pattern baldness. In all indications, specific cells are isolated, replicated into the millions and delivered back into the patient to reactivate a stalled healing process. These products utilize the Company’s proprietary manufacturing and cell expansion platform. RepliCel is also developing cell injector devices for dermal injections. In addition to addressing the delivery requirements for RCH-01 and RCS-01, they should have applications in many current dermatological injection procedures.


Teresa Leezer, CEO
Louisville, KY
RhinoCyte, Inc. is a privately owned biopharmaceutical company focused on developing transformational technologies that will change the way neurodegenerative disorders are treated. Founded in 2005, the Company is advancing innovative treatment paradigms that leverage proprietary cell therapies for high unmet clinical needs including Spinal Cord Injury (SCI), Parkinson’s Disease (PD), Amyotrophic Lateral Sclerosis (ALS) Multiple Sclerosis (MS) and Type 1 Diabetes. RhinoCyte leverages novel regenerative therapies to extend and enhance the quality of human life without immunosuppression or genetic manipulation.


Curt Herberts, Senior Director, Corporate Development & Strategy
Richmond, CA
Sangamo is a clinical-stage biopharmaceutical company focused on the research, development and commercialization of engineered DNA-binding proteins for the development of novel therapeutic strategies for unmet medical needs. The Company is a worldwide leader in the development of a proprietary technology platform that enables specific regulation of gene expression and gene modification. The basis of this platform is a naturally occurring class of transcription factors, zinc finger DNA-binding proteins (ZFPs) which can be engineered to drive desired therapeutic outcomes. Engineered ZFPs can be linked to functional domains to create ZFP transcription factors (ZFP TFs) capable of turning genes on or off. The Company can also link ZFPs to nuclease domains to create zinc finger nucleases (ZFNs) which enable precise gene-editing in cells. Engineered ZFNs can modify a cell’s DNA at a precise location, thereby facilitating correction or disruption of a specific gene or the targeted addition of a new DNA sequence.


Jill Helms, Ph.D., Professor of Surgery
Stanford, CA
(Academic Institution)
At Stanford, the goal is to discover new ways to remain fit and vigorous, well into old age. The university has identified and characterized a potent stem cell activator, WNT and shown that it activates both embryonic and adult stem cells in a wide range of assays. With CIRM funding, the university has developed a strategy for activating a patient’s own bone stem cells to reinvigorate the body’s natural regenerative process. The end result is rapid healing, back to the levels appreciated in youth.


Martin McGlynn, President & CEO
Newark, CA
StemCells, Inc. is engaged in research and clinical development of cell-based therapeutics using the Company’s platform technology, a proprietary highly purified population of human neural stem cells, HuCNS-SC® cells. The Company is developing therapies for central nervous system disorders. The Company completed enrollment in a Phase I/II clinical trial in spinal cord injury and has reported positive data for the first eight patients. The Company is also conducting a Phase I/II clinical trial in dry age-related macular degeneration (AMD). Both studies release final results next year. StemCells, Inc. is pursuing preclinical studies in Alzheimer’s disease with funding support from the California Institute of Regenerative Medicine (CIRM). In a Phase I clinical trial in Pelizaeus-Merzbacher disease (PMD), a fatal childhood myelination disorder, the Company showed preliminary evidence of donor-derived myelination in all four patients. StemCells, Inc. also markets stem cell research products including media and reagents under the SC Proven® brand.


Michael Leek, Ph.D., CEO
Edinburgh, Scotland
Commercializing the anti-cancer cytotoxicity exhibited by γδ T cells, TC BioPharm is developing an autologous therapy which involves expansion of the cells over a two-three week period to formulate a treatment for a wide-variety of different tumor types. The manufacturing process comprises several steps. The first involves isolation of peripheral blood mononuclear cells; the second involves selection/activation of γδ T cells; the final stage requires culture expansion in a GMP environment, providing therapeutically relevant cell numbers prior to administration. Although there are many different approaches to T cell immunotherapy, the process has a compelling clinical safety and efficacy record, demonstrated by a cohort of published case histories. Moreover, when contrasted with novel immunotherapies such as PDL-1 and CTLA-4 inhibitors being developed by Roche and Bristol Myers Squibb respectively, use of γδ T cells may have a complimentary and synergistic therapeutic benefit.


Sven Lee, Global Director, Sales & Business Development, Cell Processing
Lakewood, CO
(4543: Tokyo)
Terumo BCT, a global leader in blood component, therapeutic apheresis and cellular technologies, is the only company with the unique combination of apheresis collections, manual and automated whole blood processing, and pathogen reduction coupled with leading technologies in therapeutic apheresis and cell processing. Terumo believes in the potential of blood to do even more for patients than it does today. This belief inspires the Company’s innovation and strengthens Terumo’s collaboration with customers. Terumo BCT -Unlocking the Potential of Blood.


Eduardo Bravo, CEO
Leuven, Belgium
(NYSE Euronext: TIG)
TiGenix was founded in 2000 to clinically develop and bring to market novel cell therapeutic products. In 2009, the Company became the first European company to obtain centralized marketing authorization for an Advanced Therapeutic Medicinal Product (ATMP) with its Chondrocelect product, which is currently on the market in various countries in Europe. The Company’s merger with Cellerix in 2011 secured long-term growth through a clinical-stage pipeline of allogeneic adult Mesenchymal Stem Cells (MSCs). The most advanced product of this pipeline is poised to deliver Phase III results in 2015. The Company has been listed on NYSE Euronext Brussels since March 2007. Roughly 70% of the outstanding shares represent free-float, with the remaining shares being in the hands of stable reference shareholders.


Donald Kohn, M.D., Professor
Los Angeles, CA
(Academic Institution)
Sickle cell disease may be treated with allogeneic hematopoietic stem cell transplantation (HSCT), but is limited by the need to find a suitable matching donor and immunological risks. Stem cell gene therapy can allow an autologous HSCT to be done using the patient’s own bone marrow stem cells without the immune complications. We have developed a lentiviral vector that adds to hematopoietic stem cells a modified version of human beta-globin that prevents sickling of red blood cells. A Phase I clinical trial funded by CIRM Disease team Awards (DR1-01452 and DR3-06945) will be initiated in 2014 to assess feasibility, safety and secondary end-points of efficacy. Stem cell gene therapy may provide a safe and improved therapy for sickle cell disease.


Paul Laikind, Ph.D., President & CEO
San Diego, CA
ViaCyte, a private company in the field of regenerative medicine, is currently focused on developing a novel cell therapy for the treatment of diabetes. The Company’s lead product candidate, VC-01, is based on the production of pancreatic progenitors derived from human pluripotent stem cells. These cells are implanted in a durable and retrievable encapsulation device called the Encaptra® drug delivery system. Once implanted and matured, these cells are designed to secrete insulin and other regulatory factors in response to blood glucose levels. ViaCyte is headquartered in San Diego, California with additional operations in Athens, Georgia. The Company is funded in part by the California Institute for Regenerative Medicine (CIRM) and the Juvenile Diabetes Research Foundation (JDRF).


Jeff Goater, VP, Business Development
Cambridge, MA
Voyager Therapeutics is developing life-changing gene therapies for fatal and debilitating diseases of the central nervous system. The Company’s founders include scientific and clinical leaders in the fields of AAV gene therapy, expressed RNA interference and neuroscience. Voyager’s management team has deep expertise and a track record of building exceptional life science companies. The Company has developed a product engine that may transform treatment for a wide range of diseases. Voyager’s pipeline includes VY-AADC01 for Parkinson’s disease, VY-SOD101 for a monogenic form of ALS and VY-FXN01 for Friedreich’s ataxia. Voyager is committed to advancing the field of AAV gene therapy by innovating and investing in areas such as vector optimization and engineering, dosing techniques, as well as process development and production. Voyager is a private company launched in February 2014 with $45 million in Series A venture capital financing from Third Rock Ventures.