ABEONA THERAPEUTICS
Tim Miller, Ph.D., President & CEO
Dallas, TX
(NASDAQ: ABEO)
PlasmaTech Biopharmaceuticals/Abeona Therapeutics is focused on developing and delivering gene therapy and plasma-based products for severe and life-threatening rare diseases. PlasmaTech’s lead program is a gene therapy for Sanfilippo syndrome (MPS IIIA and IIIB) in collaboration with patient advocate groups, researchers and clinicians. In addition, the company is pursuing two additional proprietary platforms, Salt Diafiltration (SDF™) Process and Polymer Hydrogel Technology (PHT™), and is active in the development and commercialization of human plasma-derived therapeutics, including its proprietary alpha-1 protease inhibitor, SDF Alpha™. The company has developed a robust product pipeline that includes two commercial stage products, MuGard® and ProctiGard™, with additional follow-on products in development. www.plasmatechbio.com
ARGOS THERAPEUTICS
Shawn Leland, Pharm.D., Director, Corporate Development & Strategy
Durham, NC
(NASDAQ: ARGS)
Argos Therapeutics is a company focused on developing personalized immunotherapy products for cancer and infectious disease. The Company’s lead program is AGS-003 which is currently undergoing a Phase III study called ADAPT in front-line mRCC. www.argostherapeutics.com
ASTERIAS BIOTHERAPEUTICS
Jane Lebkowski, Ph.D., President of R&D
Menlo Park, CA
(OTCBB: ASTY)
Asterias Biotherapeutics is a leading biotechnology company in the emerging field of regenerative medicine. The Company’s core technologies center on pluripotent stem cells, which are characterized by the ability to become all cell types in the human body. Asterias is focused on developing therapies based on pluripotent stem cells to treat diseases or serious injuries in several medical areas where there is high unmet medical need and without adequate available therapies. Asterias’ two therapeutic programs, AST-OPC1 (oligodendrocyte progenitor cells) for spinal cord injuries and AST-VAC2 (antigen-presenting allogeneic dendritic cells) for lung cancer, are based on the Company’s proprietary technology platforms of Pluripotent Stem Cells and Allogeneic Dendritic Cell Immunotherapy, respectively. AST-OPC1 is currently in a Phase I/IIa clinical trial. www.asteriasbio.com
AVALANCHE BIOTECHNOLOGIES
Hans Hull, Interim CEO & President
Menlo Park, CA
(NASDAQ: AAVL)
Avalanche Biotechnologies is a clinical stage company developing adeno-associated virus (AAV) gene therapy treatments. The Company’s lead program AVA-101 for wet AMD is expected to enter a Phase IIB study later this year following positive 12-month top-line Phase IIa safety data announced in June. The AVA-201 program, also for wet AMD, uses a next generation AAV vector delivery method. Beyond this, Avalanche is developing AVA-322 and AVA-323 for color vision deficiency, commonly known as red-green color blindness. Avalanche has an ongoing collaboration with Regeneron Pharmaceuticals for the discovery, development and commercialization of therapeutics for the treatment of ophthalmic diseases, including AVA-311 for Juvenile X-linked Retinoschisis. www.avalanchebiotech.com
BIOLIFE SOLUTIONS
Mike Rice, CEO
Bothell, WA
(NASDAQ: BLFS)
BioLife Solutions develops, manufactures and markets hypothermic storage and cryopreservation solutions and precision thermal shipping products for cloud-hosted logistics management applications for cells, tissues and organs. BioLife also performs contract aseptic media formulation, fill and finish services. The Company’s proprietary HypoThermosol® and CryoStor® platform of solutions are highly valued in the biobanking, drug discovery and regenerative medicine 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. BioLife’s enabling technologies provides commercial companies and clinical researchers significant improvement in shelf life and post-preservation viability and function of cells, tissues and organs. www.biolifesolutions.com
BLUEBIRD BIO
Jeffrey Walsh, COO
Cambridge, MA
(NASDAQ: BLUE)
bluebird bio is working to develop transformative gene therapy and T cell-based immunotherapy products to provide hope to patients with severe genetic diseases and cancer. www.bluebirdbio.com
BRAINSTORM CELL THERAPEUTICS
Tony Fiorino, M.D., Ph.D., CEO
Hackensack, NJ
(NASDAQ: BCLI)
BrainStorm Cell Therapeutics is a biotechnology company developing innovative, autologous stem cell therapies for highly debilitating neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS) and Parkinson’s Disease (PD). www.brainstorm-cell.com
CALADRIUS BIOSCIENCES
David Mazzo, Ph.D., VP & CEO
Basking Ridge, NJ
(NASDAQ: CLBS)
Caladrius is a cell therapy leader with late-stage clinical programs based on a proprietary platform technology for immuno-oncology, as well as additional platform technologies for ischemic repair and immunomodulation, and capabilities that support other cell therapy developers. This integrated approach supports the industry in bringing significant life-improving medical treatments to market. www.caladriusbiosciences.com
CAPRICOR THERAPEUTICS
Linda Marbán, Ph.D., CEO
Beverly Hills, CA
(NASDAQ: CAPR)
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 one 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. www.capricor.com
CELLECTIS
André Choulika, Ph.D., CEO
Paris, France
(NASDAQ: CLLS; EURONEXT PARIS: ALCLS)
Cellectis is a preclinical stage biopharmaceutical company focused on developing immunotherapies based on gene edited engineered CAR T cells (UCART). The Company’s mission is to develop a new generation of cancer therapies based on engineered T cells. Cellectis capitalizes on its 15 years of expertise in genome engineering – based on its flagship TALEN® products and meganucleases and pioneering electroporation PulseAgile technology – to create a new generation of immunotherapies. CAR technologies are designed to target surface antigens expressed on cells. Using its life science-focused, pioneering genome-engineering technologies, Cellectis’ goal is to create innovative products in multiple fields and with various target markets. www.cellectis.com
CELL THERAPY LIMITED
Ajan Reginald, CEO
Wales, UK
(Private)
Nobel Laureate Professor Martin Evans and Ajan Reginald, the former Global Head of Emerging Technologies at Roche, founded Cell Therapy Limited (CTL) in 2009. CTL’s mission is to develop regenerative medicines for life – to discover and develop regenerative medicines to treat life-threatening and life-altering diseases. The first product from the Company’s technology platform, HeartcelTM has successfully shown in Phase II to regenerate the heart in end-stage heart failure patients, improving heart function, decreasing mortality and improving patients’ quality of life. The Company applies its extensive regenerative medicine patent portfolio and expertise to areas of high unmet clinical need. CTL has additional clinical and preclinical programs in tendon injury, dermatology, oncology, diabetes and liver fibrosis. www.celltherapyltd.com
CELYAD
Christian Homsy, M.D., CEO
Mont-Saint-Guibert, Belgium
(NASDAQ: CYAD; EURONEXT BRUSSELS: CARD)
Celyad is a leader in engineered cell therapy with clinical programs initially targeting indications in cardiology and oncology. Founded in 2007 and based in the Walloon region of Belgium, Celyad leverages research collaborations in the U.S. with the Mayo Clinic and Dartmouth College. The Company’s lead product candidate in cardiology is C-Cure®, an autologous stem cell therapeutic using adult guided stem cells for the condition of ischemic heart failure. The Company’s lead product candidate in oncology is NKG2D CAR T Cell, an autologous CAR T cell product candidate using NKG2D, a natural killer cell receptor designed to target ligands present on numerous cancer cells, including ovarian, bladder, breast, lung and liver cancers, as well as leukemia, lymphoma and myeloma. www.celyad.com
CESCA THERAPEUTICS
Ken Harris, President
Rancho Cordova, CA
(NASDAQ: KOOL)
Cesca Therapeutics is engaged in the research, development and commercialization of cell-based therapeutics for use in regenerative medicine. The Company focuses in three target markets: cellular therapeutics, medical/diagnostic devices and cell manufacturing and banking. www.cescatherapeutics.com
CYFUSE BIOMEDICAL
Koji Kuchiishi, President & CEO
Tokyo, Japan
(Private)
Cyfuse is a Tokyo-based start-up founded in 2010 to develop its proprietary technology to assemble living cells in three dimensions. Its unique bio 3D printer REGENOVA enables scaffold-free fabrication of cellular aggregates into any desired shape. The Company’s main business activities include the sales of Regenova, research and development of 3D tissue products, and subcontract manufacturing of 3D tissue products. Its technology makes it possible to produce thicker and more elastic tissue than was conventionally possible, so it is expected to bring breakthroughs in drug discovery research and regenerative medicine in a wide range of therapeutic areas. www.cyfusebio.com
DISCGENICS
Flagg Flanagan, Chairman & CEO
Salt Lake City, UT
(Private)
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 it to derive many doses from each adult tissue donation. DiscGenics’ first product, Injectable Discogenic Cell Therapy (IDCT), is in late preclinical development for the treatment of moderate degenerative disc disease. This cellular treatment offers a cost-effective and non-surgical solution to patients whom 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 in addition to follow-on indications for IDCT for adjacent level to fusion degeneration and cervical degenerative disease. www.discgenics.com
EDITAS MEDICINE
Sandra Glucksmann, Ph.D., COO
Cambridge, MA
(Private)
Editas Medicine is a genome editing company committed to unlocking the potential of the CRISPR/Cas9 technology to develop a novel class of human therapeutics that enable precise and corrective molecular modification to treat the underlying cause of a broad set of genetic diseases. The Company is focused on developing a robust CRISPR/Cas9 platform technology that is able to generate a diversified pipeline of genomic medicines to address a broad set of genetically determined diseases. www.editasmedicine.com
FATE THERAPEUTICS
Christian Weyer, M.D., President & CEO
San Diego, CA
(NASDAQ: FATE)
Fate Therapeutics is a clinical-stage biopharmaceutical company engaged in the development of programmed cellular therapeutics for the treatment of severe, life-threatening diseases. The Company’s approach utilizes established pharmacologic modalities, such as small molecules, to program the fate and function of cells ex vivo. The Company’s lead product candidate, PROHEMA®, is an ex vivo programmed hematopoietic cellular therapeutic, which is currently in clinical development for the treatment of hematologic malignancies and rare genetic disorders in patients undergoing hematopoietic stem cell transplantation (HSCT). The Company is also using its proprietary induced pluripotent stem cell platform to develop ex vivo reprogrammed hematopoietic and myogenic cellular therapeutics. www.fatetherapeutics.com
FIBROCELL SCIENCE
David Pernock, Chairman & CEO
Exton, PA
(NASDAQ: FCSC)
Fibrocell Science is an autologous cell and gene therapy company focused on developing first-in-class treatments for rare and serious skin and connective tissue diseases with high unmet medical needs. Fibrocell’s most advanced product candidate, azficel-T, uses its proprietary autologous fibroblast technology and is in a Phase II clinical trial for the treatment of chronic dysphonia resulting from vocal cord scarring. In collaboration with Intrexon Corporation (NYSE: XON), a leader in synthetic biology, Fibrocell is also developing gene therapies for skin diseases using gene-modified autologous fibroblasts. Fibrocell has submitted an IND application to the FDA for FCX-007, its lead orphan gene therapy product candidate, for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Fibrocell is in preclinical development of FCX-013, its second gene therapy product candidate, for the treatment of linear scleroderma. www.fibrocellscience.com
GENVEC
Doug Brough, Ph.D., Chairman & Chief Scientific Officer
Gaithersburg, MD
(NASDAQ: GNVC)
GenVec is a clinical-stage biopharmaceutical company with an entrepreneurial focus on leveraging its proprietary adenovector gene delivery platform to develop a pipeline of cutting-edge therapeutics and vaccines. The Company is a pioneer in the design, testing and manufacture of adenoviral-based product candidates that can deliver on the promise of gene-based medicine. GenVec’s lead product candidate, CGF166, is licensed to Novartis and is currently in a Phase I/II clinical study for the treatment of hearing loss and balance disorders. In addition to the Company’s internal and partnered pipeline, GenVec also focuses on opportunities to license its proprietary technology platform, including vectors and production cell lines, for the development and manufacture of therapeutics and vaccines to the biopharmaceutical industry. www.genvec.com
HEMOSTEMIX
Rahul Sarugaser, Ph.D., VP, Corporate Development
Calgary, Alberta
(TSX VENTURE: HEM)
Hemostemix is a small cap Phase II clinical-stage company. The Company is developing an autologous cell therapy platform technology that is based on a simple draw of a patient’s own peripheral blood followed by a proprietary manufacturing process. To date, the product has been tested in 45 clinical trial patients and more than 400 compassionate use patients. Currently, the Company is running a Phase II clinical trial to test the product for treatment of critical limb ischemia (CLI), a severe form of peripheral artery disease with poor treatment options that affects more than 10 million people in North America and Europe. The Company is also developing its platform technology for treatment of a variety of other ischemic diseases. www.hemostemix.com
HISTOGENICS
Adam Gridley, President & CEO
Waltham, MA
(NASDAQ: HSGX)
Histogenics is a regenerative medicine company focused on developing and commercializing products in the musculoskeletal segment of the marketplace. The Company’s regenerative medicine platform combines expertise in cell processing, scaffolding, tissue engineering, bioadhesives and growth factors to provide solutions that can be utilized individually or in concert to treat musculoskeletal-related conditions. Histogenics’ first investigational product candidate, NeoCart®, leverages the Company’s platform to provide an innovative treatment in the orthopedic space, specifically cartilage damage in the knee. The Company is currently enrolling its Phase III clinical trial of NeoCart in the U.S. via a BLA pathway, and covered under a Special Protocol Assessment (SPA). The Company is also developing next-generation allogeneic therapies with Intrexon Corporation (NASDAQ: XON). www.histogenics.com
INOVIO PHARMACEUTICALS
Niranjan Sardesai, Ph.D., Chief Operating Officer
Plymouth Meeting, PA
(NASDAQ: INO)
Inovio is revolutionizing the fight against cancer and infectious diseases. The Company’s immunotherapies uniquely activate best-in-class immune responses to prevent and treat disease, and have shown clinically significant efficacy with a favorable safety profile. With an expanding portfolio of immune therapies, the Company is advancing a growing preclinical and clinical-stage product pipeline. Partners and collaborators include Roche, MedImmune, University of Pennsylvania, DARPA, GeneOne Life Science, Drexel University, NIH, HIV Vaccines Trial Network, National Cancer Institute, U.S. Military HIV Research Program and the University of Manitoba. www.inovio.com
INVIVO THERAPEUTICS
Tom Ulich, M.D., Chief Scientific Officer
Cambridge, MA
(NASDAQ: NVIV)
InVivo Therapeutics is a research and clinical-stage 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, Sc.D., Professor at Massachusetts Institute of Technology, and Joseph P. Vacanti, M.D., who then was at Boston Children’s Hospital and who now is affiliated with Massachusetts General Hospital. In 2011, the Company earned the David S. Apple Award from the American Spinal Injury Association for its outstanding contribution to spinal cord injury medicine. In 2015, the Company’s investigational Neuro-Spinal Scaffold received the 2015 Becker’s Healthcare Spine Device Award. www.invivotherapeutics.com
JCYTE
Henry Klassen, M.D., Ph.D., Founder
Irvine, CA
(Private)
jCyte is a recently founded start-up company, spun out from the University of California, Irvine, to license IP relating to the derivation and use of retinal progenitor cells for treatment of blinding retinal diseases, particularly retinitis pigmentosa (RP) and age related macular degeneration (AMD), as well as to develop and commercialize this technology. The approach being tested comprises a simple intravitreal injection of allogeneic cells, without immunosuppressants. In partnership with the California Institute for Regenerative Medicine (CIRM), the National Institutes of Health (NIH) and National Center for Advancing Translational Sciences (NCATS), preclinical studies have been completed, an IND has been filed and Phase I/IIa clinical trials in RP are now underway. www.jcyte.com
JUVENTAS THERAPEUTICS
Rahul Aras, Ph.D., President & CEO
Cleveland, OH
(Private)
Juventas Therapeutics is a private, clinical stage biotechnology company developing novel non-viral gene therapies that activate natural processes to repair the body. The Company’s product candidate, JVS-100, is a non-viral gene therapy that expresses stromal cell-derived factor-1, or SDF-1, a naturally occurring signaling protein that has been shown to recruit the body’s own stem cells and promote tissue repair in a broad range of disease states. Juventas’ therapeutic approach is based on research originating from the laboratory of co-founder Marc Penn, M.D., Ph.D., FACC and the technologies he discovered and developed at the Cleveland Clinic. Juventas is currently planning Phase IIb studies in advanced ischemic heart failure and late-stage peripheral artery disease. www.juventasinc.com
KIADIS PHARMA
Manfred Ruediger, Ph.D., President & CEO
Amsterdam, The Netherlands
(Private)
Kiadis Pharma is a clinical-stage biopharmaceutical company focused on research, development and future commercialization of cell-based immunotherapy products for the treatment of blood cancers and inherited blood disorders. Kiadis Pharma believes that its innovative products have the potential to address the current risks and limitations connected with allogeneic hematopoietic stem cell transplantation (HSCT) and make HSCT a first choice treatment for blood cancers and inherited blood disorders, thereby meeting a significant unmet medical need with its products. www.kiadis.com
MAXCYTE
Douglas Doerfler, President & CEO
Gaithersburg, MD
(Private)
MaxCyte specializes in cell modification technologies to enable the discovery, development, manufacturing and delivery of innovative therapeutic products. MaxCyte is pioneering rapid expansion free mRNA-based CAR therapies through its relationship with Johns Hopkins Cancer Center. MaxCyte is enabling seven mRNA Chimeric Antigen Receptor (CAR) human solid tumor and blood cancer clinical studies including those being led by Dr. Carl June at the University of Pennsylvania and Dr. Dario Campana. mRNA expands CAR therapies to solid tumors, and manages both B cell aplasia and cytokine storm toxicities. The MaxCyte® GT™ Flow Transfection System, MaxCyte’s clinical cell loading platform is supported by U.S. FDA Master File and used in over a dozen clinical trials including seven with mRNA and gene editing of stem and immune cells. MaxCyte STX and VLX systems are being used by most of the top 20 biopharmas for small molecule drug discovery, protein development and vaccine manufacturing. www.maxcyte.com
MEDIPOST AMERICA
Antonio Lee, Ph.D., CEO & Managing Director
Rockville, MD
(Private)
MEDIPOST’s research and development is focused on providing novel therapeutics using allogeneic umbilical cord blood-derived stem cells for Osteoarthritis (OA), Broncho-pulmonary Dysplasia (BPD) and Alzheimer’s Disease (AD). MEDIPOST’s flagship product CARTISTEM® for OA has been approved by the regulatory authority (Ministry of Food and Drug Safety) in Korea as an allogeneic cell therapy product in 2012, and over 2,300 patients have been treated on market. CARTISTEM® also has successfully completed a Phase I/IIa trial in the U.S. PNEUMOSTEM® for the prevention of BPD (with orphan drug designations in Korea, U.S. and E.U.) is awaiting results from the Phase II and Phase I/II clinical trials in Korea and the U.S., respectively. NEUROSTEM® for AD is in a Phase IIa clinical trial in Korea after successfully completing a first-in-human Phase I trial with patients suffering from advanced-stage AD. MEDIPOST America is a wholly-owned U.S. subsidiary of MEDIPOST Co. (KOSDAQ: 078160). www.medipostamerica.com
MESOBLAST
Donna Skerrett, M.D., Chief Medical Officer
Melbourne, Australia
(ASX: MSB; USOTC: MBLTY)
Mesoblast Limited is a global leader in regenerative medicine. The Company has leveraged its proprietary technology platform, which is based on specialized cells known as mesenchymal lineage adult stem cells, to establish a broad portfolio of late stage product candidates. Mesoblast’s allogeneic or ‘off-the-shelf’ cell product candidates target significantly advanced stages of diseases where there are highly unmet medical needs, including cardiovascular conditions, orthopedic disorders, immunologic/inflammatory disorders and oncology/hematology conditions. The lead therapeutic product candidates under investigation include MPC-150-IM for chronic congestive heart failure; MPC-06-ID for chronic discogenic low back pain, MSC-100-IV for acute graft versus host disease, and MPC-300-IV for biologic refractory rheumatoid arthritis and diabetic nephropathy. www.mesoblast.com
MIMEDX GROUP
Marietta, GA
(NASDAQ: MDXG)
MiMedx® is an integrated developer, manufacturer and marketer of patent protected regenerative biomaterial products and bioimplants processed from human amniotic membrane. “Innovations in Regenerative Biomaterials” is the framework behind the Company’s mission to provide physicians with products and tissues to help the body heal itself. MiMedx’s biomaterial platform technologies include AmnioFix® and EpiFix®, tissue technologies processed from human amniotic membrane that is derived from donated placentas. Through the Company’s donor program, mothers delivering full-term Caesarean section births can elect in advance of delivery to donate the placenta in lieu of having it discarded as medical waste. The Company processes the human amniotic membrane utilizing its proprietary PURION® process, to produce a safe and effective allograft. MiMedx is the leading supplier of amniotic tissue, having supplied hundreds of thousands of allografts to date for application in the wound care, surgical, sports medicine, ophthalmic and dental sectors of healthcare. www.mimedx.com
MOLMED
Gian-Paolo Rizzardi, M.D., General Manager, R&D & Operations
Milan, Italy
(MLM.MI)
MolMed S.p.A. is a biotechnology company focused on research, development and clinical validation of novel anticancer therapies. MolMed’s pipeline includes two antitumor therapeutics in clinical development: TK, a cell-based therapy enabling bone marrow transplants from partially compatible donors, in absence of post-transplant immune-suppression, in Phase III in high-risk acute leukaemia; NGR-hTNF, a novel vascular targeting agent, in Phase III in malignant pleural mesothelioma and in Phase II in six more indications: colorectal, lung (small-cell and non-small-cell), liver and ovarian cancer and soft tissue sarcomas. MolMed also offers top-level expertise in cell and gene therapy to third parties to develop, conduct and validate projects from preclinical to Phase III trials, including scale-up and cGMP production of clinical-grade viral vectors, and manufacturing of patient-specific genetically-engineered cells. MolMed is headquartered at the San Raffaele Biomedical Science Park in Milan, Italy. www.molmed.com
OPEXA THERAPEUTICS
Neil Warma, President & CEO
The Woodlands, TX
(NASDAQ: OPXA)
Opexa Therapeutics is a publicly traded biotechnology company developing personalized immunotherapies with the potential to treat major illnesses, including multiple sclerosis (MS) as well as other autoimmune diseases, such as neuromyelitis optica (NMO). These therapies are based on Opexa’s proprietary T cell technology. The Company’s leading therapy candidate, Tcelna®, is a personalized T cell immunotherapy that is in a Phase IIb clinical development program (the Abili-T trial) for the treatment of secondary progressive MS. For the treatment of NMO, OPX-212 is in the preclinical stage and is based off Opexa’s existing T cell platform: antigen identification, T cell expansion and selective targeting of pathogenic T cells. OPX-212 is specifically tailored to each patient’s immune response to aquaporin-4. www.opexatherapeutics.com
ORGANOVO
Keith Murphy, CEO
San Diego, CA
(NYSE MKT: ONVO)
At Organovo, the company designs and creates functional human tissues with its breakthrough three-dimensional bioprinting technology. Organovo’s focus is on developing a range of human tissue and disease models for preclinical research and therapeutic application. The Company has partnerships with leading biopharmaceutical and academic partners across two lines of business: developing fully human, architecturally correct, 3D in vitro tissue and disease models offering significantly greater preclinical predictability; and creating novel therapeutic tissues that replace or augment native tissue function. The Company’s platform enables micro-architectural tissue features not typically seen with other platforms, and is highly flexible with respect to the cellular inputs as well as the output format. Headquartered in San Diego, Organovo was named a 2015 Technology Pioneer by the World Economic Forum and one of MIT Technology Review’s 50 most innovative global companies of 2012. www.organovo.com
ORTHOCELL
Paul Anderson, M.D., CEO
Perth, Australia
(ASX: OCC)
Orthocell is a leading musculoskeletal regenerative medicine company engaged in addressing unmet clinical needs in soft tissue regeneration. The Company’s core focus is a stem cell approach to regeneration of degenerate tendon tissue and the development of biological scaffolds for soft tissue regeneration and repair. Ortho-ATI™ is a Therapeutic Goods Administration-licensed autologous, homologous, cell-based therapy suitable for patients from elite athletes to domestic and repetitive work injuries. Ortho-ATI™ is the first truly effective, durable therapy, which addresses the underlying pathology of tendinopathy. A recently published study showed that Ortho-ATI™ treatment resulted in improved clinical function and MRI tendinopathy scores for up to five years post-treatment. CelGro™ is Orthocell’s complementary collagen device for tissue repair and regeneration. It is intended for orthopaedic, dental and general surgical applications and is an effective biological scaffold for endogenous cells and carrier matrix for delivery of cell therapies. CelGro™ is earmarked for regulatory submission in 2015. www.orthocell.com.au
OSIRIS THERAPEUTICS
Lode Debrabandere, Ph.D., President & CEO
Columbia, MD
(NASDAQ: OSIR)
Osiris Therapeutics is the world leader in researching, developing and marketing regenerative medicine products that improve health and lives of patients and lower overall healthcare costs. Having developed the world’s first approved stem cell drug, the Company continues to advance its research and development in biotechnology by focusing on innovation in regenerative medicine – including bioengineering, stem cell research and viable tissue based products. Osiris has achieved commercial success with products in orthopedics, sports medicine and wound care. Currently, Osiris’ product portfolio includes, BIO4™, a viable bone matrix that possesses all four characteristics involved in bone repair and regeneration, Cartiform®, a viable osteochondral allograft for articular repair and Grafix®, a cryopreserved placental membrane that is designed for direct application to acute and chronic wounds. www.osiris.com
OXFORD BIOMEDICA
Peter Nolan, Chief Business Officer
Oxford, U.K.
(LON: OXB)
Oxford BioMedica is a leading cell and gene-based biopharmaceutical company developing innovative medicines to improve the lives of patients with high unmet medical needs. The Company has established platform technologies in gene delivery and immunotherapy, protected by an extensive intellectual property estate. 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 is also widely used in the ex-vivo cell therapy arena. Oxford BioMedica’s product portfolio is focused on high value, growing markets. These include gene therapy products engineered to treat Parkinson’s disease (preclinical, Phase I/II), Motor Neuron Disease (preclinical) and ocular diseases (preclinical), especially retinal diseases (Phase I and Phase I/II). The Company also has MHRA certified GMP manufacturing capability to produce Advanced Medicinal Products (AMP). In particular its focus, via OXB Solutions, is on viral manufacturing and development. www.oxfordbiomedica.co.uk
PLURISTEM THERAPEUTICS
Zami Aberman, Chairman & CEO
Haifa, Israel
(NASDAQ: PSTI)
Pluristem Therapeutics is a leading developer of placenta-based cell therapy products. The Company has reported robust clinical trial data in multiple indications for its patented PLX (PLacental eXpanded) cells. The cells release a cocktail of therapeutic proteins in response to inflammation, ischemia, hematological disorders and radiation damage. PLX cell products are grown using the Company’s proprietary three-dimensional expansion technology. PLX cells are “off-the-shelf”, requiring no tissue matching prior to administration. Pluristem has a strong intellectual property position, Company-owned, GMP-certified manufacturing and research facilities, strategic relationships with major research institutions and a seasoned management team. www.pluristem.com
REGENERATIVE PATCH TECHNOLOGIES
Dennis Clegg, Ph.D., Co-Founder
Menlo Park, CA
(Private)
Regenerative Patch Technologies is a new company whose focus is the development of stem cell based therapies for ocular disease. Led by Mark Humayun, David Hinton, Dennis Clegg and Jane Lebkowski, the Company is a spin-off from academic research at the University of Southern California (USC), California Institute of Technology (Caltech), University of California, Santa Barbara (UCSB) and City of Hope, and is funded by the California Institute for Regenerative Medicine (CIRM). Current efforts are aimed at developing a therapy for geographic atrophy/dry age-related macular degeneration. This disease is caused by loss of the retinal pigmented epithelium (RPE), which leads to death of rods and cones. The product (CPCB-RPE1) consists of a sheet of RPE derived from human embryonic stem cells on a parylene scaffold, which will be surgically implanted into the eye. An IND application was recently cleared by the FDA for a Phase I/IIa study. www.regenerativepatch.com
REGENXBIO
Karen Kozarsky, Ph.D., VP, R&D
Rockville, MD
(Private)
REGENXBIO is a leading biotechnology company focused on the development, commercialization and licensing of recombinant adeno-associated virus (AAV) gene therapy. The Company’s foundation is its proprietary AAV gene delivery platform, the NAV® Technology Platform. REGENXBIO’s mission is to develop and bring to market best-in-class gene therapy treatments for a range of severe diseases with significant unmet medical needs using the NAV Technology Platform. The Company seeks to accomplish this mission through a combination of internal development efforts and the efforts of third-party licensees. The Company’s most advanced internally developed candidates include programs for the treatment of severe genetic diseases including Mucopolysaccharidosis Type I (MPS I). REGENXBIO also has a program for wet age-related macular degeneration (wet AMD) that is in the preclinical stage. The Company plans to build internal gene therapy franchises in the metabolic, neurodegenerative and retinal therapeutic areas. www.regenxbio.com
RENEURON
Olav Hellebo, CEO
Guildford, U.K.
(LON: RENE)
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 diseases 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 ischaemia are in clinical development and its cell-based treatment for the blindness-causing disease, retinitis pigmentosa, is about to enter the clinic in the U.S. ReNeuron is also advancing a proprietary platform technology to exploit nanoparticles (exosomes) secreted by stem cells as potential new drug candidates targeting a range of cancers. www.reneuron.com
REPLICEL
David Hall, President & CEO
Vancouver, B.C.
(OTCMKTS: REPCF)
RepliCel is a regenerative medicine company focused on developing autologous cell therapies that address diseases caused by a deficit of healthy cells required for normal healing and function. The Company’s RCT-01, RCS-01 and RCH-01 cell therapies are designed to treat chronic tendinosis, UV-damaged or aged skin and pattern baldness. Shiseido has an exclusive license for RCH-01 in certain Asian countries including Japan, China and South Korea. All product candidates are based on RepliCel’s innovative technology utilizing cell populations isolated from a patient’s own healthy hair follicles. The Company is also developing a propriety injection device optimized for the administration of its products and licensable for use with other dermatology applications. The Company’s product pipeline is comprised of multiple clinical trials anticipated to launch through 2015, in addition to Shiseido’s own clinical trial of RCH-01 and the device in late prototype development. www.replicel.com
SANGAMO BIOSCIENCES
Elizabeth Wolffe, Ph.D., VP, Corporate Communications
Richmond, CA
(NASDAQ: SGMO)
Sangamo BioSciences is a clinical-stage gene therapy company focused on Engineering Genetic Cures™ for monogenic and infectious diseases by deploying its novel DNA-binding protein technology platform in therapeutic genome editing and gene regulation. The Company has a Phase II clinical program to evaluate the safety and efficacy of a novel T cell based ZFP Therapeutic® for the treatment of HIV/AIDS (SB-728). This application is being expanded into hematopoietic stem cells to enable the genetic engineering of an HIV-resistant immune system. Phase II clinical studies for this stem cell approach are expected to begin in 2015. Sangamo’s other therapeutic programs are focused on monogenic diseases such as hemoglobinopathies, including sickle cell disease and beta-thalassemia, and additional monogenic indications including hemophilia A and B, lysosomal storage disorders and Huntington’s disease. www.sangamo.com
STEMBIOSYS
Robert Hutchens, President & CEO
San Antonio, TX
(Private)
StemBioSys is a privately-held biomedical company focused on the isolation, growth and delivery of adult stem cells for research, therapeutic or drug discovery applications. Its patented and proprietary technology platforms – licensed from the University of Texas Board of Regents – overcome key obstacles to creating clinically useful stem cell therapies. The centerpiece of these enabling technologies is the HPMETM (High Performance Micro Environment™) platform. This cell derived 3D microenvironment allows a variety of stem cells to replicate more rapidly and with greater preservation of stem cell properties compared to more traditional tissue culture substrates. This disruptive technology is positioned to transform and improve the methods and cost of isolating, growing and delivering various stem cell populations for applications in research, therapeutic and drug discovery. Earlier this year the Company launched its first product directed to the stem cell research market, BM – HPME™. www.stembiosys.com
STEMCELLS, INC.
Martin McGlynn, President & CEO
Newark, CA
(NASDAQ: STEM)
StemCells, Inc. is developing its HuCNS-SC® platform technology (purified human neural stem cells) as a treatment for disorders of the central nervous system. Top line data from the Phase I/II clinical trial in thoracic spinal cord injury (SCI) showed measurable gains involving multiple sensory modalities and segments, including conversion of two of seven patients with complete injuries (AIS A) converting to incomplete injuries (AIS B). StemCells, Inc. has completed its Phase I/II clinical trial in geographic atrophy of age-related macular degeneration (GA-AMD), the most severe form of dry AMD and the leading cause of blindness in the elderly. Based upon interim results for patients who had completed 12 months of follow-up, the Company reported reductions in the rate of progression in GA as compared to the untreated eye and the expected natural history of the disease. StemCells, Inc. has initiated controlled proof-of-concept Phase II studies in both SCI and AMD. www.stemcellsinc.com
TAIWAN BIO
Shih-Chieh Hung, M.D., Ph.D., Principal Investigator
Taipei City, Taiwan
(Private)
Taiwan Bio was founded in July 2014, in partnership with a research team at Taipei Veterans General Hospital and National Yang Ming University. Dr. Shih-Chieh Hung’s team developed a patented technology for allogeneic mesenchymal stem cell therapy. Taiwan Bio has completed several preclinical tests, showing the successful application of the patented technology on ischemia limb, bone reconstruction, cartilage regeneration, and Achilles tendon rupture. The same technology has also been tested in the application of allogeneic mesenchymal stem cells for the treatment of GvHD, autoimmune diseases and musculoskeletal disorders. The Phase I/IIa clinical trial of ischemic limb was launched in 2015, and is expected to be completed in one to two years. The Company’s advanced technology will provide a novel and effective treatment for diseases involving an aged society, in order to improve the quality of life for patients and their families. www.taiwan-bio-thera.com
TERUMO BCT
Dave Flaten, VP, Cell Processing
Tokyo, Japan
(NYSE MKT: TSE)
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. The Company believes in the potential of blood to do even more for patients than it does today. This belief inspires Terumo BCT’s innovation and strengthens the Company’s collaboration with customers. As Terumo BCT strives to make even safer, higher-quality transfusions available to more people, the Company can unlock the potential of blood and cell therapies. Terumo BCT can help customers bring even more treatment options to patients through advanced blood therapies. The Company can support researchers in developing cellular therapies that may fundamentally improve healthcare. Terumo BCT is guided by customers’ needs, aspirations and ongoing drive to improve efficiency and patient outcomes. www.terumobct.com
TIGENIX
Eduardo Bravo, CEO
Leuven, Belgium
(Euronext Brussels: TIG)
TiGenix is an advanced biopharmaceutical company focused on developing and commercializing novel therapeutics from its proprietary platform of allogeneic, expanded adipose-derived stem cells in inflammatory and autoimmune diseases. www.tigenix.com
TISSUEGENE
Moon Jong Noh, Ph.D., VP, R&D
Rockville, MD
(Private)
TissueGene focuses on a cell-mediated gene therapy in regenerative medicine. The Company’s lead product, TG-C, is for the treatment of symptomatic osteoarthritis of the knee. TissueGene has completed a Phase II trial and Phase III trials will begin early next year. In Korea, TG-C is near completion of its Phase III clinical trials with an expected launch date of 2016. The patented approach to providing relief for osteoarthritis has significant advantages over current technologies. As a single injection therapy consisting of allogeneic cells, it is convenient for patients (no rehabilitation), simple for doctors to use in outpatient settings and mass-producible. Above all, in clinical trials involving nearly 220 patients, TG-C has been proven to provide long-term pain and functional relief for nearly 24 months, longer than any product currently on the market. www.tissuegene.com
UNIQURE
Jörn Aldag, President & CEO
Amsterdam, The Netherlands
(NASDAQ: QURE)
uniQure is delivering on the promise of gene therapy through single treatments with potentially curative results. The Company has developed a modular platform to rapidly bring new disease-modifying therapies to patients with severe disorders. uniQure is engaged in multiple partnerships and has obtained regulatory approval of its lead product, Glybera, in the European Union for a subset of patients with lipoprotein lipase deficiency (LPLD). www.uniqure.com
VERICEL
Nick Colangelo, CEO
Cambridge, MA
(NASDAQ: VCEL)
Vericel Corporation (formerly Aastrom Biosciences) is the leader in developing patient-specific expanded cellular therapies for use in the treatment of patients with severe diseases and conditions. The Company markets two cell therapy products in the U.S., Carticel® (autologous cultured chondrocytes), an autologous chondrocyte implant for the treatment of cartilage defects in the knee, and Epicel® (cultured epidermal autografts), a permanent skin replacement for the treatment of patients with deep dermal or full thickness burns comprising greater than or equal to 30% of total body surface area. Vericel is also developing MACI, a third-generation autologous chondrocyte implant for the treatment of cartilage defects in the knee, and ixmyelocel-T, a patient specific multicellular therapy for the treatment of advanced heart failure due to ischemic dilated cardiomyopathy (DCM). www.vcel.com
VIACYTE
Paul Laikind, Ph.D., President & CEO
San Diego, CA
(Private)
ViaCyte is a privately held regenerative medicine company focused on developing a novel cell therapy for the treatment of diabetes. ViaCyte is conducting a Phase I/II clinical trial of the Company’s lead VC-01 product candidate in patients with type 1 diabetes who have minimal to no insulin-producing beta cell function. ViaCyte’s VC-01’s combination product candidate is based on the production of pancreatic progenitor cells derived from human pluripotent stem cells. These progenitor cells are implanted in a durable and retrievable encapsulation device. Once implanted and matured, these cells are designed to secrete insulin and other regulatory factors in response to blood glucose levels. The VC-01 product candidate is being developed as a potential long-term diabetes treatment without immune suppression, and without risk of hypoglycemia or other diabetes-related complications. www.viacyte.com
VOYAGER THERAPEUTICS
Jeff Goater, SVP, Finance & Business Development
Cambridge, MA
(Private)
Voyager Therapeutics is a gene therapy company developing life-changing treatments for fatal and debilitating diseases of the central nervous system (CNS). Voyager is committed to advancing the field of AAV (adeno-associated virus) gene therapy through innovation and investment in vector engineering and optimization, dosing and delivery techniques, as well as process development and production. The Company’s pipeline is focused on CNS diseases in dire need of effective new therapies, including Parkinson’s disease, a monogenic form of amyotrophic lateral sclerosis, Friedreich’s ataxia and Huntington’s disease. Voyager has broad strategic collaborations with Genzyme, a Sanofi company, and the University of Massachusetts Medical School. Voyager was founded by scientific and clinical leaders in the fields of AAV gene therapy, expressed RNA interference and neuroscience. www.voyagertherapeutics.com