2013 COMPANY PRESENTERS
52 Company Presentations featuring 75+ Unpartnered Products from 100+ Clinical Stage Programs
The 3rd Annual Regen Med Partnering Forum included a pipeline loaded with partnering opportunities. Collectively, the 52 companies that presented at the program are conducting over 100 clinical trials and have over 75 product candidates seeking partnerships – not to mention their 30+ products already on the market!
2013 Company Presenters
Nick Colangelo, President & CEO
Founded in 1989 and with headquarters in Ann Arbor, Michigan, Aastrom Biosciences is dedicated to the development of stem cell treatments for critical cardiovascular diseases. Aastrom is currently evaluating its autologous cellular therapies in US clinical trials in the treatment of dilated cardiomyopathy (DCM). These critical diseases are associated with significant morbidity and mortality and very limited treatment options.
Robert Brenner, M.D., President & CEO
AlloCure, Inc. is a pioneer in the development of cell therapies for the treatment of kidney disease and a leader in the acute kidney injury (AKI) field. The company is developing AC607, an allogeneic mesenchymal stem cell therapy for the treatment of AKI. Building on the success of a Phase I clinical trial, AlloCure is conducting ACT-AKI, a randomized, multicenter, double-blinded, placebo-controlled Phase II trial in cardiac surgery subjects with post-operative AKI. Currently, there are no available treatment options for patients with AKI other than supportive care. AC607 represents the first application of a mesenchymal stem cell therapy to alter the course of this life threatening condition.
Gil Van Bokkelen, Ph.D., Chairman & CEO
Athersys is a clinical stage biotechnology company engaged in the discovery and development of therapeutic product candidates designed to extend and enhance the quality of human life. The company is developing its MultiStem® cell therapy treatment, a patented, adult-derived “off-the-shelf” stem cell platform for disease indications in the cardiovascular, neurological, inflammatory and immune disease areas. The company currently has five clinical stage programs involving MultiStem, including for treating inflammatory bowel disease (partnered with Pfizer), ischemic stroke, damage caused by myocardial infarction, and for the prevention of graft versus host disease. Athersys has also developed a diverse portfolio that includes other technologies and product development opportunities, and has forged strategic partnerships and collaborations with leading pharmaceutical and biotechnology companies, as well as world-renowned research institutions in the United States and Europe to further develop its platform and products.
William Dolphin, Ph.D., CEO
(ASX: AVH; OTCQX: AVMXY)
Avita Medical is a regenerative medicine global medical technology company. Avita’s patented and proprietary tissue-culture, collection and application technology provides innovative treatment solutions derived from a patient’s own skin. The company’s lead product, ReCell® Spray-On Skin™, is used in a wide variety of burns, chronic wounds, plastic, reconstructive and cosmetic procedures. ReCell is patented, CE-marked for Europe, TGA- registered in Australia, and SFDA-cleared in China. FDA trials on the safety and efficacy of ReCell for use in burns and scars are in progress in the US.
Lesley Stolz, Ph.D., EVP of Corporate Development
(NYSE MKT: BTX)
BioTime is a biotechnology company focused on the emerging field of regenerative medicine. Our core technologies center on novel stem cells capable of becoming all of the cell types in the human body. Products made from these “pluripotent” stem cells are being developed by our subsidiaries, each of which concentrates on different medical specialties, including: neuroscience, oncology, orthopedics, and blood and vascular diseases. BioTime’s commercial strategy targets near-term commercial opportunities such as Hextend and our current line of research products: ACTCellerate cell lines and associated ESpan culture media, HyStem hydrogels, and GMP human embryonic stem cell lines. In addition, the initiation of clinical testing of ReneviaTM as a cell delivery device is expected in 2013, and the initiation of clinical validation testing of PanC-Dx as a novel blood-based cancer screen is expected during 2014.
Faraz Ali., VP of Commercial Development
Bluebird bio is developing next generation products based on the transformative potential of gene therapy to treat patients with severe genetic and orphan diseases. The company has two clinical stage products in development for childhood cerebral adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell disease, a preclinical oncology program in partnership with Celgene, a world-class team and a broadly applicable gene therapy platform.
BRAINSTORM CELL THERAPEUTICS
Adrian Harel, Ph.D., Director of R&D
BrainStorm is a biotechnology company developing innovative adult stem cell therapies derived from autologous bone marrow cells for the treatment of neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS). NurOwn™, the company’s novel stem cell technology, differentiates the patient’s mesenchymal stem cells (MSC) into neurotrophic factor (NTF)-secreting cells (“MSC-NTF”) that are transplanted into the spine and/or muscle tissue. These neuron-supporting cells aim to protect existing motor neurons, promote their growth and re-establish nerve-muscle interaction. The ability to differentiate mesenchymal stem cells into MSC-NTF cells, and confirmation of their activity and potency before transplantation, makes NurOwn a first-of-its-kind approach for treating neurodegenerative diseases. BrainStorm is currently conducting a Phase IIa clinical trial at the Hadassah University Medical Center in Jerusalem. In parallel, BrainStorm is planning to launch a multi-center Phase II ALS trial in the US in late 2013, pending FDA approval.
Linda Marbán, Ph.D., Founder & CEO
Los Angeles-based Capricor is a biotechnology company that aims to create powerful, yet easy-to-administer cardiac stem cell treatments to regenerate damaged heart muscle and improve heart function for patients having suffered a heart attack. Using proprietary technology, Capricor is working to develop and commercialize therapies that utilize stem cells that are indigenous to the heart itself. Where most other organizations derive stem cells from a range of pre-cursor and non-cardiac tissues and then attempt to differentiate those cells to become and support heart tissue, Capricor’s work begins and ends with the heart. Patient recruitment is ongoing for ALLSTAR, Capricor’s Phase I/II study using allogeneic CDCs (CAP-1002) to treat patients 30-days to 1-year after a heart attack. The objective of ALLSTAR is to demonstrate that the infusion of CDCs to damaged myocardium will show a relative reduction in scar tissue.
CELLULAR DYNAMICS INTERNATIONAL
Robert Palay, Chairman & CEO
Cellular Dynamics International, Inc. (CDI) is a leading developer of stem cell technologies for in vitro use in drug discovery, toxicity testing and chemical safety; in vivo and cell-based therapeutic research; and stem cell banking. CDI harnesses its unique manufacturing technology to produce differentiated tissue cells in industrial quality, quantity and purity from any individual’s stem cell line created 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. CDI’s facilities are located in Madison, Wisconsin. CDI’s headquarters are located in Madison, Wisconsin, with a second facility in Novato, California.
Eric Ahrens Ph.D., Member, Board of Directors
Celsense is a privately held biotechnology company offering novel MRI agents that allow scientists and clinicians to monitor the location and quantity of transplanted cells. The company’s products have application in the fields of regenerative medicine, immunotherapy, and the study of inflammation and immune system response. Celsense is currently collaborating with leading pharmaceutical and biotechnology organizations and has developed products suitable for human use. Celsense was founded in 2005 to commercialize imaging platforms developed at Carnegie Mellon University.
CENTRE FOR COMMERCIALIZATION OF REGENERATIVE MEDICINE (CCRM)
Michael May, Ph.D., CEO
CCRM is a not-for-profit technology translation center focused on the development and commercialization of early-stage RM technologies. It aligns academic innovation with market needs in three ways: by assessing and in-licensing high potential academic RM discoveries, by providing core facilities and contract services to enable rapid, cost-efficient development of these technologies, and by engaging industry early and often to provide de-risked, high-potential pre-commercial products. CCRM is supported by Canada’s Centres of Excellence for Commercialization and Research (CECR) Program, as well as by a diverse Industry Consortium of thirty leading RM companies.
CORD BLOOD REGISTRY
Geoffrey Crouse, CEO
Cord Blood Registry® (CBR) is the world’s first family cord blood stem cell bank, started in 1992. CBR collects, processes and stores newborns’ cord blood and cord tissue for future medical or therapeutic use. Cord Blood Registry is headquartered in San Bruno, California. CBR owns and operates its laboratory and storage facility, located in Tucson, Arizona and currently stores more than 500,000 individual cord blood/cord tissue samples. Today, it is the largest facility dedicated and designed exclusively for cord blood stem cell processing and storage and cord tissue preservation. In partnership with CBR, cord blood stem cells are being evaluated in a series of FDA approved clinical trials as potential treatment for autism, cerebral palsy, traumatic brain injury and pediatric stroke.
CYTORI THERAPEUTICS, INC.
Doug Arm, Senior VP of Operations
Cytori Therapeutics, Inc. is developing cell therapies based on autologous adipose-derived regenerative cells (ADRCs) to treat cardiovascular disease and repair soft tissue defects. Our scientific data suggest ADRCs improve blood flow, moderate the immune response and keep tissue at risk of dying alive. As a result, we believe these cells can be applied across multiple ischemic conditions. These therapies are made available to the physician and patient at the point-of-care by Cytori’s proprietary technologies and products, including the Celution® system product family.
Flagg Flanagan, Chairman & CEO
DiscGenics is a spinal therapeutics company developing novel treatments based on tissue engineering and progenitor cell science to treat patients suffering from intervertebral disc diseases. Back pain is the second most common reason to visit the doctor and degenerative disc disease costs the US economy more than $100 billion per year in treatment and lost productivity. We believe the product we are developing, Injectable Discogenic Cell Therapy (IDCT), will regenerate the intervertebral disc itself, thereby reducing pain and delaying or even eliminating the need for painful and expensive back surgery.
David Pernock, Chairman & CEO
(NYSE MKT: FCSC)
Fibrocell Science, Inc. is an autologous cell therapy company focused on the development of innovative products for aesthetic, medical and scientific applications. Fibroblast cells produce collagen and elastin, important for the health, beauty and repair of skin, soft tissues and organs. The company’s proprietary, autologous fibroblast technology, LAVIV™ (azficel-T), is the first and only autologous cellular product approved by the FDA to improve the appearance of moderate to severe nasolabial fold (smile line) wrinkles in adults. Fibrocell is leveraging its autologous fibroblast technology and expertise with its manufacturing excellence to expand its aesthetics portfolio and to address medical applications in which fibroblasts have the potential to treat unmet medical needs, including restrictive burn scarring, vocal cord scarring, acne scarring and rare genetic collagen deficiencies, such as recessive dystrophic epidermolysis bullosa (RDEB). There are currently no FDA-approved products to treat these conditions.
Jason Gardner, D.Phil., VP of R&D, Head of Regenerative Medicine Discovery Performance Unit
GlaxoSmithKline (GSK) is a science-led global healthcare company that researches and develops a broad range of innovative medicines and brands. Our products are used by millions of people around the world, helping them to do more, feel better and live longer. We have three primary areas of business in pharmaceuticals, vaccines and consumer healthcare. Our commercial success depends on creating innovative new products and making these accessible to as many people who need them as possible. Research is vitally important to the success of our business, and we spent just under £4 billion in 2012 in our search to develop new medicines. GSK has an established global Regenerative Medicine Discovery Performance Unit in R&D which drives the discovery and development of regenerative medicines inside and outside GSK, including partnerships with The Harvard Stem Cell Institute (stem cell-based assays for drug discovery), and Telethon Institute for Gene Therapy (stem cell gene therapy for rare genetic diseases). Progress in these alliances will be presented together with the discovery and development of the approved GSK drug, Promacta, a small molecule activator of endogenous progenitor cells, as an example of new regenerative medicines.
Gail Naughton, Ph.D., Chairman & CEO
Histogen is a regenerative medicine company focused on stimulating stem cells in vivo through the administration of embryonic-like soluble and insoluble compositions secreted by hypoxia-induced multipotent stem cells in suspension cultures. The company currently uses its cell conditioned media (CCM) as an additive to anti-aging skin care products and as an injectable for hair growth. A pilot, Phase I/II, and physician sponsored clinical trial have all demonstrated safety as well as significant efficacy in hair regrowth at the 12 week and one year timepoint. A small molecular weight subfraction of the CCM induces apoptosis in over 20 human cancer cell lines. The company has a JV called PUR Biologics for use of the CCM and extracellular matrix components for orthopedic applications. One proprietary manufacturing process produces the two products simultaneously, each of which have multiple aesthetic and therapeutic applications, to create a robust product portfolio.
Henry Klassen, M.D., Ph.D., Founder
Paul Bresge, Board Advisory Chairman
jCyte is an early stage startup out of UC Irvine, tasked with licensing and commercial development of the intellectual property of Drs. Henry Klassen and Jing Yang. This primarily includes cell-based technologies for minimally invasive treatment of the blinding disease retinitis pigmentosa (RP), as well as other retinal diseases such as age-related macular degeneration (AMD). The company has received orphan status designation from the FDA for application of this technology to RP. Drs. Klassen and Yang were recently awarded a $17.1 million CIRM DT2 grant to move this project through the FDA approval process and into clinical trials.
Rahul Aras, Ph.D., President & CEO
Juventas Therapeutics is a private clinical-stage company developing novel therapies for ischemic cardiovascular disease. The company’s lead product, JVS-100, is a non-viral 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. Juventas is currently enrolling multiple Phase II clinical trials to test therapy efficacy in heart failure and critical limb ischemia patients.
Thomas Fellner, Ph.D., Director, Cell Therapy Development Services
(SWL: CH: LONN)
Lonza is one of the world’s leading suppliers to the pharmaceutical, healthcare and life science industries. Our products and services span our customers’ needs from research to final product manufacture. As a leader in cell therapy manufacturing, Lonza offers world class technology platforms in the areas of cGMP cell culture including adult and pluripotent stem cells; viral-based therapeutic manufacturing; custom biotherapeutic culture media; and a full line of custom bioassays.
Madhusudan Peshwa, EVP, Cellular Therapies
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.
Antonio Lee, Ph.D., Associate Director
MEDIPOST operates the largest private cord blood bank in Korea with 12 years of R&D experience using human Umbilical Cord Blood-derived Mesenchymal Stem Cells (hUCB-MSCs) with successful clinical development and commercialization of allogeneic stem cell products. The world’s first allogeneic stem cell drug named CARTISTEM® received its Biologics License Application (BLA) from the Ministry of Food and Drug Safety (formerly known as KFDA) in Korea in January 2012. CARTISTEM® treats knee cartilage defects including osteoarthritis and leads to regeneration of hyaline-like cartilage. Since launched in May 2013, over 600 patients have been treated in the Korean market. PNEUMOSTEM® – another hUCB-MSC drug in the pipeline, helps prematurely born infants form developing Bronchopulmonary Dysplasia (BPD) and is in a Phase II clinical trial in Korea with an orphan-drug designation. NEUROSTEM® for Alzheimer’s disease has successfully completed its Phase I clinical trial and a Phase II trial will commence soon.
Donna Skerrett, M.D., Chief Medical Officer
(ASX: MSB; USOTC: MBLTY)
Mesoblast Limited is developing biotherapeutics based on its proprietary cell-based technologies which include its adult Mesenchymal Precursor Cell (MPC) technology platform, currently used to develop products derived from bone marrow and adipose tissue sources, its Dental Pulp Stem Cells, and expanded Hematopoietic Stem Cells. Additionally, the company is developing biotherapeutics based on protein factors derived from its proprietary cellular platforms. Mesoblast’s strategic MPC product development focus is in three major and distinct areas: systemic inflammatory conditions; cardiovascular disease; and orthopedic diseases of the spine. Mesoblast is partnered with Teva for the development and commercialization of its MPC products in a number of fields, including cardiovascular diseases and neurologic conditions. Mesoblast has also established an alliance with Lonza for the clinical and long-term commercial manufacturing of its allogeneic MPC products. Its strong cash reserves enable simultaneous product development with future revenues expected from milestone payments, distribution arrangements and direct sales.
Cynthia Bamdad, Ph.D., Chairman & CEO
Minerva is focused on the space between stem cells and cancer cells. Minerva discovered that a novel growth factor receptor, MUC1*, and its ligand NM23 mediate the growth of 75% of cancers but 100% of pluripotent stem cells. Minerva elucidated the feedback loop that limits stem cell self-replication, but which cancer cells override for continuous self-replication. NM23 makes human stem cells revert to the naïve state and maintains them there indefinitely. Naïve stem cells have long been thought to be the holy grail of stem cells. We demonstrated that our naïve stem cells have a much higher cloning efficiency and differentiate in ways that are superior to primed stem cells. Minerva’s stem cell growth system is free of feeder cells, conditioned media, or other growth factors. Cells remain essentially 100% pluripotent, requiring no manual dissection or other manipulations that would interfere with large scale production and automated cell culture.
Claudio Bordignon, M.D., Chairman & CEO
(Milan Stock Exchange: MTA)
MolMed S.p.A. is a medical biotechnology company focused on research, development and clinical validation of innovative therapies to treat cancer. The MolMed approach is based on an integrated strategy that provides on the one hand drugs that effectively target reduction of the tumor mass in the acute stage and, on the other, highly selective therapies to eliminate the residual disease.
MOORES CANCER CENTER – UC SAN DIEGO
Dennis Carson, M.D., Professor Emeritus
The creation of the Highly Active Anti-Leukemia Stem Cell Therapy (HALT) program was developed at UC San Diego Moores Cancer Center. This work is funded in part by a CIRM Disease Team Research Grant. The investigators of the team include Dennis Carson, M.D., Catriona Jamieson, M.D., Ph.D., and Thomas Kipps, M.D., Ph.D. The business development team consists of Ida Deichaite, Ph.D.
Jed Johnson, Ph.D., Chief Technology Officer
Nanofiber Solutions, LLC is a medical device manufacturer developing a new class of implants for soft tissue/organ repair/regeneration and of three-dimensional (3-D) scaffold products that advance research in cellular therapeutics and drug discovery. Using our unique approach to a platform technology, we combine the benefits of synthetics and biologics while eliminating the risks, and supply chain issues inherent in biologics. Specifically, our technology is used to build scaffolds that are critical in the development of life-saving and life-changing tissue engineered implants. We manufacture the world’s first nanofiber tracheal implant. Our NanoTrachea implants have been used successfully in several European surgeries and we are rapidly expanding our product offerings and patent estate based on this proven scaffold technology.
Robin Smith, M.D., Chairman & CEO
(NYSE MKT: NBS)
NeoStem, Inc. is a leader in the emerging cellular therapy industry. Our business model includes the development of novel proprietary cell therapy products as well as operating a contract development and manufacturing organization providing services to others in the regenerative medicine industry. The combination of a therapeutic development business and revenue-generating service provider business provides the company with capabilities for cost effective in-house product development and immediate revenue and cash flow generation.
NEW YORK STEM CELL FOUNDATION
Stephen Chang, Ph.D., VP, Research & Development
The New York Stem Cell Foundation (NYSCF) is an independent organization founded in 2005 to accelerate cures and better treatments for patients through stem cell research. NYSCF employs over 40 researchers at the NYSCF Research Institute and is an acknowledged leader in stem cell research and in developing pioneering stem cell technologies, including the NYSCF Global Stem Cell Array. NYSCF supports 60 researchers at leading institutions worldwide through its Innovator Programs, including the NYSCF – Druckenmiller Fellowships and the NYSCF – Robertson Investigator Awards. NYSCF focuses on translational research in a model designed to overcome the barriers that slow discovery and replaces silos with collaboration. NYSCF achieved six major discoveries in the field, including: the discovery of a therapy to prevent transmission of mitochondrial diseases in 2012; the derivation of the first-ever, patient-specific embryonic stem cell line in 2011; and the creation of the first disease model from iPS cells.
Geoff MacKay, President & CEO
Organogenesis Inc. is a leader in regenerative medicine, pioneering the development and commercialization of living cell-based therapies for more than two decades. Driven by its mission to bring the medical marvel of regenerative medicine to patients and standardize its use in everyday medical care, Organogenesis’ two FDA-approved commercial products represent medical breakthroughs in the field of regenerative medicine. Apligraf®, Organogenesis’ lead product, was the first mass-produced bio-engineered cell-based product to receive FDA approval (in 1998), and now with two FDA-approved indications and over 500,000 units shipped for patient use, it is one of the leading regenerative medicine brands in the world. GINTUIT™, Organogenesis’ newest product, FDA-approved in 2012, is the first and only living cell-based product specifically designed to regenerate soft tissue in the mouth.
Sharon Presnell, Ph.D., Chief Technology Officer & EVP of Research & Development
(NYSE MKT: ONVO)
Organovo designs and creates functional, three-dimensional human tissues for medical research and therapeutic applications. We are collaborating with pharmaceutical and academic partners to develop human biological disease models in three dimensions. These 3D human tissues have the potential to accelerate the drug discovery process, enabling treatments to be developed faster and at lower cost.
Francois Binette, Ph.D., VP, Research & Business Development
(NYSE MKT: BTX)
OrthoCyte Corp is the orthopedic regenerative medicine subsidiary of BioTime Inc. With nearly 40 distinct osteochondro progenitors, and a proprietary injectable settable biopolymer cell delivery system, OrthoCyte is positioned to address nearly all musculoskeletal diseases and injuries with the most robust orthobiologic platform in the industry. Our initial focus on spine will target both the early events of chronic back pain, stopping and reversing degenerative disc disease and late-stage disease with spinal fusion. Our minimally invasive approach promises to decrease procedural risks, while uniquely tailored cell therapy matching specific tissue requirements will enhance the chances of therapeutic success. OrthoCyte is currently developing bone repair solutions for market introduction within the next 24 months while advancing its cell therapy for spine and other orthopedic indications through animal studies.
OSIRIS THERAPEUTICS, INC.
Frank Czworka, General Manager of Wound Care
For 20 years Osiris Therapeutics, Inc. has focused on developing and marketing first-in-class cellular therapies for serious medical conditions. In 2005, Osiris launched the world’s first commercial stem cell product, Osteocel®, for bone regeneration. In 2012, Osiris was granted approval for the world’s first systemically infused stem cell drug, Prochymal® (remestemcel-L), which is also the first drug approved for the treatment of refractory acute graft-versus-host disease – a lethal complication of bone marrow transplantation. Osiris currently markets Prochymal, as well as Grafix® and Ovation® for wound and soft tissue repair, and Cartiform®, a viable cartilage mesh for articular cartilage injury. Our technology harnesses the intuitive power of cells to promote the body’s natural healing abilities and, in doing so, makes “Smart Medicine.® Right Now.®” a reality. Come learn more about the future.
PLURISTEM THERAPEUTICS, INC.
William Prather, M.D., Sr. VP Corporate Development
(NASDAQCM: PSTI; TASE: PLTR)
Pluristem Therapeutics Inc. is a biotechnology company that develops cell therapies derived from human placentas and is the sole manufacturer of these therapies. Pluristem uses its proprietary, three-dimensional (3D) technology platform for the efficient, controlled, mass production of its PLacental eXpanded (PLX) cell therapy product candidates that can be administered without the need for histocompatability matching. PLX cells serve as a biologically controlled drug delivery platform capable of secreting various therapeutic cytokines and other soluble factors in response to environmental signals. Pluristem’s strategy is to make PLX cells available as an intramuscular therapy for the treatment of a variety of indications that possess, as a common denominator, the inflammatory/ischemic pathophysiologic state. This includes the use of our PLX cell products involving the indications of peripheral artery disease, soft tissue orthopedic injuries, preeclampsia, acute radiation sickness, and the stimulation of hematopoiesis in diseased or injured bone marrow.
PROGENITOR CELL THERAPY
Robert Preti, Ph.D., President & Chief Scientific Officer
(NYSE MKT: NBS)
Progenitor Cell Therapy (PCT) is a Contract Development and Manufacturing Organization (CDMO) with an unprecedented 14-year track record of cell therapy-exclusive operational, quality and regulatory excellence. We are focused on supporting the full-cycle of development and commercialization of our clients’ cell therapy products by offering a comprehensive spectrum of services designed to enable and expedite cell therapy development and commercialization: cGMP and GLP manufacturing; product, process, and assay development; GTP tissue/cell sourcing, processing and banking; storage, distribution and delivery; and regulatory support and cell therapy consulting services. At PCT we recognize that each client’s needs are as unique as the cell therapy they are developing. We position ourselves as the collaborative extension of our clients’ team, and use our resources and expertise as the building blocks of a tailored service platform, designed to address the clients’ specific needs, while allowing them control and freedom to utilize all of their existing resources and expertise.
Eric Halioua, CEO
Promethera® Biosciences’ mission is to develop cell therapy products to treat liver diseases using stem cells. The company is a spin-off from the Laboratory of Paediatric Hepatology and Cell Therapy in Brussels, Belgium. Promethera Biosciences develops a new cell therapy product using patented allogeneic stem cells expanded from healthy human liver tissue. This product called HepaStem aims to treat a wide variety of liver genetic and acquired diseases affecting children and adults. The innovation resides in both the simplicity of the treatment, which does not require radical surgery (compared to liver transplantation), and in the wide variety of liver pathologies that can be addressed with the same product. This product has received two orphan drug designations from the EC and the FDA for indications. In conjunction, the company is developing HepaScreen, a unique cell model for the pharmaceutical industry to mimic metabolism and detoxification of new drugs by the human liver.
Deborah Eppstein, Ph.D., President & CEO
Q Therapeutics is developing a novel proprietary glial cell therapy product (Q-Cells®, human glial restricted progenitor cells and their glial cell progeny) for treatment of neurodegenerative diseases of the CNS. Q-Cells produce both astrocytes and oligodendrocytes, the support cells that enable neuronal health and function in the CNS. Our first clinical indication is treatment of patients with ALS, with clinical trials targeted to commence in 2014. We also are evaluating Q-Cells in preclinical studies in disease models relevant to transverse myelitis, multiple sclerosis and spinal cord injury. Q has a strong intellectual property estate arising from the work of Dr. Mahendra Rao as well as developed internally by Q, with 16 issued patents and more pending, providing protection for Q-Cells as well as other neural lineage cell types.
Teresa Leezer, President
RhinoCyte™, Inc. is a biopharmaceutical company focused on developing an innovative stem cell platform for neurodegenerative diseases. The technology will be leveraged to change the paradigm on how Spinal Cord Injury is treated. Our mission is to become a leader in the advancement of cell based therapies by providing innovative stem cell solutions to treat a variety of unmet needs in the neurodegenerative arena including Spinal Cord injuries (SCI), Parkinson’s disease (PD), Amyotrophic Lateral Sclerosis (ALS), and Multiple Sclerosis (MS). The RhinoCyte™ technology affords patients a novel regenerative therapy without immunosuppression or genetic manipulation. RhinoCyte’s™ lead product is an autologous progenitor cellular replacement termed RhinoCytes™ for SCI. The second product is targeted toward patients with Parkinson’s disease.
Aidan Courtney, CEO
Roslin Cells is a world leader in the production of clinical and research grade pluripotent stem cells for use in therapy and research. The Company is engaged in all stages of the cell therapy development process and is working with commercial and academic research groups in the development of novel cell therapies. The company couples its multidisciplinary expertise with the operation of a major state of the art clean room facility located in the Edinburgh BioQuarter which enables it to produce GMP grade cells for clinical trials. The company applies the highest quality standards and achieves its goals in a robust, ethical manner compliant with current and expected regulatory requirements.
SANFORD-BURNHAM MEDICAL RESEARCH INSTITUTE
Evan Snyder, Ph.D., M.D., Professor, Del E. Webb Center for Neuroscience, Aging, and Stem Cell Research; Director, Stem Cells and Regenerative Biology Program, Sanford-Burnham Medical Research Institute
Sanford-Burnham Medical Research Institute is firmly positioned in the upper echelon of elite laboratory biomedical research. In only 35 years, the Institute has established itself as a preeminent research institute employing more than 1,200 people (including 900 scientists) dedicated to furthering the understanding of health and disease. Our chief product is knowledge, dispensed in the more than 330 research publications generated annually by the Institute’s teams of scientists and shared with the world to advance science and medicine. For the past decade (1999-2009), Sanford-Burnham ranked number one worldwide among all organizations in biology and biochemistry for the impact of its research publications according to the Institute for Scientific Information. Sanford-Burnham’s unique, highly collaborative culture has propelled its research programs in cancer, neurodegeneration, diabetes, and infectious, inflammatory and childhood diseases, as well as its world-class capabilities in stem cell research and drug discovery, into the top tier of independent medical research organizations.
SANGAMO BIOSCIENCES, INC.
Elizabeth Wolffe, Ph.D., Senior Director, Corporate Communications
Sangamo BioSciences, Inc. is focused on research and development of novel DNA-binding proteins for therapeutic gene regulation and genome editing. The company has ongoing Phase II clinical trials to evaluate the safety and efficacy of a novel ZFP Therapeutic® for the treatment of HIV/AIDS. Sangamo’s other therapeutic programs are focused on monogenic diseases, including hemophilia, Huntington’s disease and hemoglobinopathies such as beta-thalassemia and sickle cell anemia. Sangamo’s core competencies enable the engineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs). Engineering of ZFPs that recognize a specific DNA sequence enables the creation of sequence-specific ZFP Nucleases (ZFNs) for gene modification and ZFP transcription factors (ZFP TFs) that can control gene expression and, consequently, cell function.
Evan Facher, Ph.D., President & CEO
SironRX Therapeutics is a clinical-stage company developing novel therapies for dermal wound repair with a specific focus on mitigating scar formation and accelerating wound healing. The company’s lead product, JVS-100 is a clinically tested, non-viral DNA plasmid that encodes stromal cell-derived factor-1 (SDF-1). SDF-1 is naturally produced by the human body in response to tissue injury activating the body’s own native repair processes through recruiting endogenous stem cells and other progenitor cells to the damaged region. The company is evaluating JVS-100 drug safety and its ability to reduce scar formation and accelerate wound repair in a double-blind, placebo-controlled, randomized clinical trial for surgical wounds. Previous clinical studies with JVS-100 have demonstrated the drug is well tolerated and safe.
STANFORD CARDIOVASCULAR INSTITUTE
Joseph Gold, Ph.D., Assistant Director, Translational Research
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.
Jill Helms, D.D.S., Ph.D., Professor, Department of Surgery
Dr. Jill Helms is actively working with a disease team on a WNT-based therapy targeting endogenous stem cells to enhance skeletal healing in the elderly. WNT proteins are naturally-occurring signaling molecules that guide stem cell fate and tissue regeneration. The therapeutic potential of WNT proteins has been recognized for years but challenges in manufacturing, formulation, and delivery have hindered their development as drug candidates. Our technology addresses these obstacles and we have accrued strong proof-of-concept data that a WNT-based approach is broadly effective at activating endogenous stem cells for tissue regeneration, especially in elderly patients.
Peter Savas, CEO
StemBioSys Inc. (SBS) is developing technologies in the stem cell space which have broad applications across stem cell research, therapeutic applications and drug discovery. The core technology revolves around a natural biologic substrate or extracellular matrix which we have shown has significant advantages over standard culture procedures for stem cell isolation and expansion. Research on this substrate has revealed enhancement of proliferation of stem cells from a host of different sources which is indicative of the enabling nature of this technology. Use of this ECM has allowed SBS researchers to identify and expand a rare subset of stem cells found in human umbilical cord blood. SBS has identified and is investigating other opportunities to expand this core technology into many different areas of research and clinical applications. Through both organic and partnering efforts, we intend to further develop these technologies leading to therapeutic applications that will add significant clinical value.
Martin McGlynn, President & CEO
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. The company’s lead product candidate, HuCNS-SC® cells (purified human neural stem cells), is currently in development for a broad range of central nervous system disorders. In a Phase I clinical trial in Pelizaeus-Merzbacher disease (PMD), a fatal myelination disorder in children, the company has shown preliminary evidence of donor-derived myelination in all four patients transplanted with HuCNS-SC cells. The company is conducting a Phase I/II clinical trial in chronic spinal cord injury in Switzerland and has reported positive data for the first three patients. The company is also conducting a Phase I/II clinical trial in dry age-related macular degeneration (AMD), and is pursuing preclinical studies in Alzheimer’s disease. StemCells also markets stem cell research products, including media and reagents, under the SC Proven® brand.
Chris Moore, Director, Cell Processing
Terumo BCT, a global leader in blood component 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. We believe in the potential of regenerative medicine to do even more for patients than it does today. This belief inspires our innovation and strengthens our collaboration with customers. As we strive to make even safer, higher-quality innovative products available to more people, we can unlock the potential of blood and cell therapies. We can support researchers in developing cellular therapies that may fundamentally improve healthcare. Terumo BCT is guided by our customers’ needs, aspirations and ongoing drive to improve efficiency and patient outcomes.
Tom Smart, Chairman & CEO
TheraBiologics is a clinical stage biopharmaceutical company developing human neural stem cell (NSC)-mediated cancer therapies. Its NSC platform delivers potent anti-cancer agents selectively to invasive cancer sites, minimizing off target toxicities. An initial clinical safety study for lead product candidate TBX01 has been completed in recurrent brain tumor patients. All three lead product candidates, TBX01, TBX02, and TBX03, are expected to be the subject of ongoing clinical trials in 2014. Research programs have been funded by grant monies and private foundations, including an $18 million Disease Team award from CIRM to TheraBiologics’ founder, Dr. Aboody, to develop TBX02 for clinical brain tumor trials. Dr. Aboody has also recently been awarded an NIH-NINDS Translational grant to develop TBX02 for clinical application for metastatic neuroblastoma by 2017.
Eduardo Bravo, Managing Director & CEO
(NYSE Euronext: TIG)
TiGenix was founded in the year 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. The merger with Cellerix in 2011 secured long term growth through a clinical stage pipeline of allogeneic adult Mesencgymal Stem Cells (MSCs), which today comprises a Phase III, a Phase Ib/IIa and a Phase I program in the inflammatory and autoimmune disease areas. These operations are supported by a dedicated commercial and manufacturing infrastructure. TiGenix is now a fully integrated company with commercial infrastructure in Western Europe in place, and state of the art manufacturing facilities in Sittard-Geleen (Netherlands) and Madrid (Spain). The company has been listed on NYSE Euronext Brussels since March 2007.
UNIVERSITY OF UTAH CELL THERAPY & REGENERATIVE MEDICINE FACILITY
Jo-Anna Reems, Ph.D., Scientific Director
The University of Utah Cell Therapy and Regenerative Medicine Facility (CTRM) consistently provides safe, high quality products for therapeutic and research applications. Our goal is to facilitate the availability of cellular and tissue based therapies to patients by bridging efforts in basic research, bioengineering and the medical sciences. To achieve this goal, the CTRM has assembled the expertise and infrastructure to address the complex regulatory, financial and manufacturing challenges associated with delivering innovative cell and tissue-based products to patients. The CTRM has 15 years of experience assisting with IND applications to the FDA and with manufacturing and delivering hematopoietic stem cells, mesenchymal stromal cells, human embryonic stem cells, glial restricted progenitors and other tissue-specific derived progenitors.
Hina Chaudhry M.D., Founder
VentriNova, Inc. is a biotechnology company focused on developing treatments for cardiovascular disease based on the use of cyclin A2 to induce cardiac regeneration after myocardial infarction or in heart failure. VentriNova’s unique approach for heart regeneration does not rely on stem cell transplantation, rather on stimulating the endogenous fetal gene program that causes heart muscle cells to divide. This pathway is dormant after birth in mammals, and the lack of significant proliferative capacity of heart muscle cells in adults underlies the extensive mortality and morbidity of heart disease. VentriNova, Inc. was awarded a small business grant from the NIH and obtained the highest in the nation for NIH NHLBI small business grants over a 5 year period. Initial venture investment in VentriNova is through Broadview Ventures. VentriNova was chosen by TEDMED 2013 as one of the 50 most innovative healthcare companies globally this year.
Eugene Brandon, Ph.D., Director, Strategic Relations and Project Management
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-protective encapsulation medical device (Encaptra® drug delivery system). Once implanted, the precursor cells mature into endocrine cells that secrete insulin and other hormones in a regulated manner to control blood glucose levels. ViaCyte’s goal is a product that can free patients with type 1 and type 2 diabetes from long-term insulin dependence. ViaCyte has received substantial financial support from both the California Institute for Regenerative Medicine (CIRM) and Juvenile Diabetes Research Foundation (JDRF).