Conference Co-Hosts

ARM_220The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 275 members and is the leading global advocacy organization in this field. www.alliancerm.org

Sponsor the Program

Interested in gaining extensive exposure to over 1,000 leading decision makers in the cell and gene therapy field? Contact Laura Parsons at lparsons@alliancerm.org for more information on sponsorship opportunities and raise your profile at this year’s conference.

SSSCC-Logo_220The Sanford Stem Cell Clinical Center at UC San Diego Health exists to transform the enormous potential of stem cell science into real therapies for patients. Our goal is to advance therapies and cures by supporting ethically conducted clinical trials that result from scientific discoveries. The Sanford Stem Cell Clinical Center was created to protect and counsel patients, and accelerate innovative stem cell research into patient diagnostics and therapy. As the center’s clinical arm, the CIRM Alpha Stem Cell Clinic at UC San Diego Health works to accelerate the design of innovative stem cell research to advance the testing and delivery of safe and effective stem cell-based therapies in regenerative medicine. Our current clinical trials focus on diabetes, spinal cord injuries, heart problems and cancer. https://health.ucsd.edu/clinicaltrials/sanford/Pages/default.aspx

Platinum Sponsor

PCT is the global cell therapy manufacturing and development service platform of the Hitachi Chemical Regenerative Medicine Business Sector, leveraging nearly two decades of PCT platform experience exclusively focused on the cell therapy industry. The PCT service platform includes contract development and manufacturing organization (CDMO) services, at current Good Manufacturing Practices (cGMP) standards, including clinical manufacturing, commercial manufacturing and manufacturing development.

Hitachi Chemical Advanced Therapeutics Solutions (HCATS), is a wholly owned subsidiary of Hitachi Chemical Company, Ltd. (Hitachi Chemical) representing Hitachi Chemical’s Regenerative Medicine Business Sector in the U.S. HCATS’ vision is a world in which transformative cell-based therapeutics are accessible to all, and its mission is to provide its clients with high-quality, scalable, innovative, reliable and cost-efficient manufacturing platforms and services, such as the PCT platform, to advance commercialization of cellular therapies. Robert Preti, Ph.D., the CEO and President of HCATS is the General Manager of the Hitachi Chemical Regenerative Medicine Business Sector. www.pctcelltherapy.com

Program Partners

FIRM_155x68The Forum for Innovative Regenerative Medicine (FIRM) is a Japanese incorporated association whose mission is to promote regenerative medicine. FIRM’s goal is to establish a stable and welcoming business environment to benefit patients both in Japan and the rest of the world. FIRM works closely with the government to establish legislative, regulatory and business environments that are the most suitable for the practice of regenerative medicine. FIRM also works with academia to accelerate the transfer of basic research into commercialization. The organization is confident that these groups will foster biotechnology in the medical industry, along with the new regulatory and business environments established in Japan. FIRM was formed in 2011 and has more than 220 members of Japanese companies with expertise in diverse fields such as pharmaceutics, biotechnology, manufacturing, transportation, insurance and related supporting areas for regenerative medicine. FIRM organizes ISO/TC 276 (Biotechnology) in Japan, as the first and only incorporated association in this field in Japan. Further, newly established FIRM-CoNCEPT (Committee for Non-Clinical Safety Evaluation of Pluripotent Stem Cell-derived ProducT) and MEASURE (Multisite Evaluation Study on Analytical Methods for Non-Clinical Safety Assessment of HUman-Derived REgenerative Medical Products) define a roadmap and best practices for internationally acceptable concepts/consensus, and the testing methods for reduction of tumorigenicity risk of cell-based products with a particular focus on pluripotent stem cell-derived products. These attempts are expected to propel Japan several steps ahead of the rest of the world in regenerative medicine. FIRM’s main office is located in Tokyo. https://firm.or.jp/en

Sanford-Consortium_250The mission of the Sanford Consortium for Regenerative Medicine is to advance stem cell research through collaborative, multi-disciplinary interactions. This collaboratory enables scientists from the La Jolla Institute for Allergy and Immunology, the Salk Institute for Biological Studies, the Sanford Burnham Prebys Medical Discovery Institute, The Scripps Research Institute, and the University of California, San Diego to work side-by-side in a facility specifically designed to achieve breakthrough discoveries. Consistent with the objectives of the California Institute for Regenerative Medicine (CIRM), Sanford Consortium for Regenerative Medicine researchers are applying the powers of stem cells to promote diagnoses, treatments and cures for degenerative diseases and injuries. Originally assembled in March 2006 as the San Diego Consortium for Regenerative Medicine, the organization was renamed in September 2008 in recognition of an extraordinary gift from T. Denny Sanford. www.sanfordconsortium.org

Gold Sponsors

Audentes Therapeutics is a clinical-stage biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases. The company has four product candidates in development, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), AT342 for the treatment of Crigler-Najjar syndrome, AT982 for the treatment of Pompe disease, and AT307 for the treatment of the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia (CASQ2-CPVT). Audentes is a focused, experienced and passionate team committed to forging strong, global relationships with the patient, research and medical communities. www.audentestx.com

BlueRock_220Driven by a vision to liberate patients from the burden of degenerative disease, BlueRock Therapeutics is ushering in a new era of cell-based medicine that repairs the body when it cannot repair itself. Founded in 2016 through one of the largest Series A financings in biotech history, BlueRock and its team of preeminent scientists are pioneering cell therapies that replace dead, damaged or dysfunctional cells to restore critical natural functions in the body. Using an approach that can be applied to multiple diseases with great unmet need, BlueRock is initially targeting severe brain and heart conditions, with the goal of altering the course of disease and drastically improving quality of life. BlueRock’s culture is defined by scientific innovation, highest ethical standards and an urgency to bring transformative treatments to all who would benefit. www.bluerocktx.com

Brammer_200x68Brammer Bio is an experienced cell and gene therapy CDMO focused on process and analytical development, clinical supply and now commercial supply. With more than a decade of experience, our 240-strong, highly skilled team has managed over 100 client projects delivering first-in-human clinical materials to enable us to accelerate our client’s products from the laboratory to patients in need. We work in close collaboration with each client’s team to deliver successful programs. We are Manufacturing Personalized. Through this, we enable the delivery of novel medicines to improve patient health Helping to Cure. www.brammerbio.com

Cryoport_185Cryoport is the premier provider of cold chain logistics solutions to the life sciences industry through our purpose-built proprietary packaging, information technology and specialized logistics expertise. We provide leading-edge cold chain logistics solutions for biologic materials such as immunotherapies, stem cells, CAR-T cells and reproductive cells for clients worldwide including points-of-care, CROs, central laboratories, pharmaceutical companies, contract manufacturers and university researchers. Cryoport’s unparalleled information technology centers around our proprietary Cryoportal Logistics Management Platform, which facilitates management of the entire shipment process. Our state-of-the-art technology also includes our innovative SmartpakII Condition Monitoring System, which provides visibility of the location and the key aspects of critical shipments. The SmartpakII works in conjunction with our Cryoportal to provide real-time information reporting about shipments, integrated condition monitoring, logistics and shipper qualification performance in a single data steam. Cryoport also has a dedicated 24/7/365 customer service team to proactively monitor each shipment, allowing for invention when necessary. www.cryoport.com

Lonza_180x68Lonza is one of the world’s leading and most-trusted suppliers to the pharmaceutical, biotech and specialty ingredients markets. We harness science and technology to create products that support safer and healthier living and that enhance the overall quality of life. The company is organized into two market-focused segments: Pharma and Biotech and Specialty Ingredients. The core competencies that span these segments are advanced manufacturing and quality-control systems, superior regulatory expertise, in-depth market knowledge and extensive technical customer-support and research and development capabilities. www.lonza.com

Terumo_210Terumo 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 technologies. We believe in the potential of blood to do even more for patients than it does today. This belief inspires our innovation and strengthens our collaboration with customers. www.terumobct.com

Thermo_210x68As the world leader in serving science, Thermo Fisher Scientific is uniquely positioned to provide the quality materials, services and support needed to accelerate the pace of advanced therapy development. We understand the complexity of this rapidly evolving industry and partner with our customers as they transition from discovery to clinical research and commercial manufacturing. Our portfolio of trusted and recognized products and services, combined with over 50 years of cell culture expertise, enables us to provide comprehensive solutions to support every step of the advanced therapy process from cell collection through manufacture to clinical site delivery. Through our Thermo Scientific, Applied BioSystems, Invitrogen, Fisher Scientific, Fisher BioServices, Unity Lab Services, and Gibco Cell Therapy Systems (CTS) brands, we offer an unmatched combination of innovative technologies, purchasing convenience and comprehensive support. www.thermofisher.com

Vineti_180x68Vineti builds 21st century technology to drive the production and delivery of 21st century medicine. The company combines leading software expertise with deep, first-hand industry experience in commercializing personalized medicines to develop a cloud-based platform that ensures quality, scale, security, efficiency, traceability and safety. The Vineti platform provides actionable insights to continually optimize the cell and gene therapy process, accelerating time to revenue and decreasing costs. Caregivers and pharmaceutical pioneers are empowered to help more patients more effectively and safely, treatments are better understood and improved over time and most importantly, there’s an opportunity to provide greater health outcomes – and cures – to patients in need. Vineti was co-founded by GE and the Mayo Clinic and is based in San Francisco, CA. www.vineti.com

Silver Sponsors

Be The Match BioTherapies® partners with organizations pursuing life-saving cellular therapies in every stage of development – from discovery through commercialization. Built on the foundation established over the last 30 years by the National Marrow Donor Program® (NMDP)/Be The Match®, we have unparalleled experience managing cellular therapies. Our cell therapy supply chain delivery for autologous or allogeneic therapies is enabled by high-touch, personalized case management and a robust, customizable software platform, MatchSource®. Our experience in cell sourcing and collection allows us to provide cells consented for research, clinical or commercial use. Researchers have access to clinical trial services through our research program CIBMTR® (Center for International Blood and Marrow Transplant Research®). In addition, we have the infrastructure in place to collect and analyze patient outcomes post-cell or gene therapy treatment at the time points required by regulatory authorities. www.bethematchbiotherapies.com

Catapult_160The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 180 experts focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. www.ct.catapult.org.uk | www.gov.uk/innovate-uk

CCRM_135CCRM, a Canadian not-for-profit funded by the Government of Canada, the Province of Ontario and leading academic and industry partners, supports the development of regenerative medicines and associated enabling technologies, with a specific focus on cell and gene therapy. A network of academic researchers, leading companies, strategic investors and entrepreneurs, CCRM aims to accelerate the translation of scientific discovery into marketable products for patients with specialized teams, funding and infrastructure. CCRM sources and evaluates intellectual property from around the globe, offers various consulting services, conducts development projects with partners and establishes new companies built around strategic bundles of intellectual property. CCRM has a fully resourced 6,000 square foot development facility used to both evaluate and advance technologies, a Centre for Advanced Therapeutic Cell Technologies and a (coming soon!) GMP facility within a 40,000 square foot office designed for advanced cell manufacturing. CCRM is the commercialization partner of the Ontario Institute for Regenerative Medicine and the University of Toronto’s Medicine by Design. CCRM is hosted by the University of Toronto and was launched in Toronto’s Discovery District on June 14, 2011. www.ccrm.ca

Cell Therapies Pty Ltd (CTPL) is the leading provider of manufacturing and deployment infrastructure for cell based therapies in Australasia and SE Asia. CTPL provides contract process development and GMP manufacturing and delivery services to product development companies across all cell types. Our expertise is needle-to-needle ensuring complete product security and control and includes collection management (especially apheresis), cryopreservation, cell processing and distribution. Our home base at The Peter MacCallum Cancer Institute (our parent) in Melbourne’s Parkville biomedical precinct provides unparalleled access to clinical and research resources to enable rapid start-up or extension of clinical trials and centres of excellence for commercial deployment, all supported by Australia’s efficient Clinical Trial Notification scheme. CTPL holds numerous cGMP manufacturing licenses with Australia’s Therapeutic Goods Administration and is proud to be working with some of the world’s leading stem cell companies. An alliance with Pharmabio in Japan enables us to project our capabilities under a common quality management system and project governance structure into this important market. We welcome enquiries from new contract customers. www.celltherapies.com.au

ChemoMetec is a Danish founded company which specializes in the development, manufacturing and sales of high-quality automated Cell Counters, Advanced Cell Analyzers and Image Cytometers to help streamline research and production processes for maximum efficiency. Our instruments are based on a patented, unique technology platform that ensures a high quality of analysis results and reliability. The instruments are known for their robustness and high precision as well as the ease of use yet advanced analysis capabilities. Our primary focus is on cell counting and cell analysis, especially for use in life sciences research, clinical diagnostics and in production and quality control within the pharmaceutical industry. www.chemometec.com

Cognate Bioservices is a fully-integrated contract bioservices organization providing the highest level of scientific and management expertise. Cognate provides full development and cGMP manufacturing services to companies and institutions engaged in the development of cell-based products. The combination of highly experienced staff, cGMP facilities and an international track record makes Cognate one of the most experienced contract manufacturers of cell-based products in the world today. www.cognatebioservices.com

Evidera_200x63_updated-2018Evidera, PPD’s peri- and post-approval business unit, is a leading provider of evidence-based solutions to demonstrate the real-world effectiveness, safety and value of biopharmaceutical and biotechnology products. We help biopharmaceutical and biotechnology companies generate the evidence needed to optimize the market access and commercial potential of their products. We provide integrated and tailored scientific expertise and global operational capabilities. Our offerings include interventional studies, real-world observational research and data analytics, patient-centered outcomes studies, epidemiological studies, modeling and simulation, meta-analysis, literature reviews, market access consulting and communications and medical writing. Evidera has approximately 500 employees representing over 35 nationalities in locations around the world, with major offices across North America and Europe. Our scientific and consulting staff are methodological and thought leaders with an average of 15 years of experience, contributing to hundreds of submissions to payers and regulators, and publishing over 2,200 peer-reviewed articles dating back nearly 30 years. www.evidera.com

FHCC_180At Fred Hutchinson Cancer Research Center, home to three Nobel laureates, interdisciplinary teams of world-renowned scientists seek new and innovative ways to prevent, diagnose and treat cancer, HIV/AIDS and other life-threatening diseases. Fred Hutch’s pioneering work in bone marrow transplantation led to the development of immunotherapy, and Hutch scientists continue to be thought leaders and innovators in the field. Fred Hutch’s robust pipeline of translational therapies is cultivated and supported by over 300 faculty members, onsite vector manufacturing and cell processing facilities, stellar clinical care partners and a commitment to innovation. www.fredhutch.org

Histogenics_170_websiteHistogenics is a leading regenerative medicine company developing and commercializing novel tissue therapies that may offer more rapid and durable recoveries for patients with pain and loss of function due to musculoskeletal conditions. Histogenics regenerative medicine platform combines expertise in cell processing, scaffolding, tissue engineering and bioadhesives to create tissue ex-vivo. Histogenics first investigational product candidate, NeoCart is designed to treat cartilage defects in the knee and is currently in Phase III clinical development. NeoCart is designed to exhibit characteristics of articular, hyaline cartilage prior to and upon implantation into the knee and therefore does not rely on the body to make new cartilage. As a result, NeoCart is the only product in development or on the market with a one-year primary superiority endpoint as compared to the standard of care. There are 500,000 or more knee cartilage procedures in the U.S. each year, with many healthy active adults avoiding treatment as they seek other alternatives. Left untreated, even a small cartilage defect can expand in size and progress to debilitating osteoarthritis, ultimately necessitating a joint replacement procedure. Osteoarthritis is more common in adults over the age of 50, but the condition and precursors of the condition can be observed much earlier, and cartilage damage is believed to be one of the leading contributors of this disease. www.histogenics.com

Medpace_170_websiteMedpace is a global full-service clinical research 0rganization (CRO) providing comprehensive development services for drug, biologic and device programs with a specialized focus on advanced therapies including cell and gene therapies. Medpace has strong experience supporting development programs for regenerative medicine products across a number of therapeutic areas including cardiovascular, neuroscience, musculoskeletal and diabetes. With extensive medical expertise, a renowned regulatory affairs department, across six continents, Medpace conducts studies and navigates regulatory requirements worldwide. From feasibility, research site compatibility, safety and logistics, Medpace brings efficiencies and operational excellence to regenerative medicine development programs. In addition, Medpace offers integrated imaging and lab capabilities through its family of companies to provide cohesive, streamlined and standardized trial management. Learn more about Medpace’s focus on cellular, tissue and gene therapy medicines at www.medpace.com.

Mesoblast_180x63Mesoblast is building a leading franchise of cellular medicines to treat serious and life-threatening illnesses. The company has leveraged its proprietary disruptive technology platform, based on allogeneic off-the-shelf mesenchymal lineage adult stem cells, to target advanced stages of diseases with high, unmet medical needs including cardiovascular diseases, immune-mediated and inflammatory conditions, spine orthopedic disorders and oncology/hematology diseases. Each of these products has its own distinct technical characteristics, target indications, separate commercialization potential and unique partnering opportunities. In 2016, Mesoblast’s licensee in Japan, JCR Pharmaceuticals, received the first full approval for an allogeneic cellular medicine in Japan. www.mesoblast.com

Millipore-Sigma_150MilliporeSigma, a leader in life science, is a business of Merck KGaA, Darmstadt, Germany. Our purpose is to solve the toughest problems in life science by collaborating with the global scientific community- and through that, we aim to accelerate access to better health for people everywhere. We provide scientists and engineers with best-in-class lab materials, technologies and services. Our broad portfolio of 300,000 products along with our service offering, help tackle our customers scientific challenges. www.emdmillipore.com

MiMedx_160MiMedx is the global premier processor, marketer and distributor of human amniotic tissue. MiMedx has distributed over one million amniotic tissue grafts worldwide and achieved profound clinical outcomes in therapeutic areas including ophthalmology, spine, chronic wounds, dental, orthopedic surgery, sports medicine and urology. The company’s patented PURION Process is the foundation of the MiMedx AmnioFix and EpiFix products that serve to regenerate damaged or diseased tissues by delivering human extracellular matrix components, essential growth factors and specialized mediating cytokines. AmnioFix and EpiFix modulate inflammation, reduce scar tissue formation and enhance healing. The proprietary PURION Process dry preserves human amniotic membrane tissue, resulting in minimally manipulated tissue products that address numerous serious unmet medical needs requiring tissue regeneration or healing inside and outside the body. Through the company’s unique process for dehydrating human amniotic tissues, improved clinical outcomes, increased patient quality of life and reduced financial burden to the healthcare system are achieved. www.mimedx.com

Polyplus-transfection applies its 15+ year expertise in transfection to the development of novel transfection reagents suitable for production of virus clinical batches and use in GMP processes, such as PEIpro-HQ, the unique PEI optimized for large scale virus production. PEIpro-HQ has proven to be the gold standard for production of various virus types, providing high, reliable and extremely reproducible virus titers, thanks to a very tight quality environment. Polyplus-transfection product range also covers in vivo delivery (in vivo-jetPEI and GMP grade reagents), CRISPR delivery reagents and R&D applications. www.polyplus-transfection.com

SCM Lifescience was established with the vision of developing treatment options for difficult to treat diseases, to provide a new source of hope for patients and to contribute to the welfare of humanity as a whole using proprietary stem cell isolation technology and stem cell production technology. Through over 10 years of research and development, SCM Lifescience has obtained intellectual property protection for its high-purity stem cell isolation technology – registered patent in Korea in 2008, U.S. in 2010, Japan in 2012, China in 2013 and the EU in 2014; high-purity stem cell manufacturing technology – U.S. patent in 2014; and treatment of graft-versus-host disease – patents from Japan in 2014, U.S. in 2015 and the EU in 2016. Based on these technical breakthroughs, high-purity stem cell therapy shall be provided to treat various immune diseases such as graft-versus-host disease, acute pancreatitis, atopic dermatitis and Type I diabetes. SCM’s research will also focus on the field of regenerative medicine for technological breakthroughs including the regeneration of skin, cartilage, bone, hair growth and other tissues and organs. www.scmlifescience.com/en

StemBioSys_190_websiteStemBioSys, based in San Antonio, Texas, is a privately-held biomedical company focused on enhancing the growth and delivery of adult stem cells for research, therapeutic or drug discovery applications. Its patented and proprietary technology platforms – licensed from the UT Systems – overcome key obstacles to creating clinically useful stem cell therapies. Our technologies represent the next evolution in cell research and are branded to the research market under the CELLvo™ label. The centerpiece of these enabling technologies is our CELLvo™ Matrix. This cell derived 3-dimensional microenvironment allows a variety of stem cell types to replicate more rapidly, maintain a small cell size and express markers indicative of retained stem cell potency beyond that seen with traditional tissue culture substrates. Additionally the CELLvo™ Matrix has been shown to enhance tissue specific differentiation of various primary and iPS cell types. The company recently announced a Xeno Free version of its core cell expansion technology. The company also has several cell products available for the research market. The cell products are all isolated and expanded on our CELLvo™ Matrix. We are actively seeking collaboration partners interested in applying our matrix and/or cell technologies in a variety of applications. www.stembiosys.com

Synpromics is a synthetic biology company with IP and know-how in the design of synthetic promoters. Our synthetic promoters enable the controlled expression of genes at a specific place, in a specific environment, at a specific time or in response to a specific biological condition. PromPT is our proprietary technology engine to rapidly design, develop and validate novel promoter systems. Custom promoter design parameters typically include: expression level, tissue specificity, inducibility, size, kill-switch, environmental and pathologic response. We provide solutions to our customers across multiple fields of use including: Targeted gene therapies and DNA vaccines, where we have created tissue specific promoters with activity levels in excess of CMV and CBA and a size of less than 500bp; Enhanced functionality of cell therapies through creation of inducible and condition specific promoters; Viral vector bioprocessing, for which we have developed inducible promoters for the creation of stable producer cell lines for lentivirus, retrovirus and AAV manufacture; Biotherapeutic protein production, for which we have created CHO expression systems with activity levels in excess of 10X CMV. www.synpromics.com

Bronze Sponsors

AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trials in X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), and achromatopsia (ACHM CNGB3 and ACHM CNGA3). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, adrenoleukodystrophy (ALD), which is a disease of the central nervous system (CNS), and otology. The clinical-stage XLRS and XLRP programs, the discovery program in ALD and two additional ophthalmology programs are being developed in collaboration with Biogen. In addition to its product pipeline, AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products. www.agtc.com

Akron_118x57Akron Biotech is an innovative biotechnology company with a strategic focus on supplying GMP-qualified ancillary materials and services to the regenerative medicine industry. Akron manufactures a range of products for cell therapy discovery, development and commercialization. These products include growth factors, sera and purified proteins, custom-made cell culture media, tailored scaffolds with biomaterials and proprietary cryopreservation formulations. In addition, our services and capabilities such as raw materials qualification, logistics and packaging optimization, bioassay design, validation and regulatory services are one of the many opportunities for Akron to provide our customers with unique knowledge and expertise. As a regulatory compliant company (ISO certified), Akron supports clients with rigorous documentation and quality standards to fulfill their regulatory demands. Our unique capabilities allow us to seamlessly transition from R&D to preclinical and clinical development with minimal change control, and thus drive the emerging regenerative medicine sector to unmet clinical needs through affordable and seamless manufacturing options. www.akronbiotech.com

Athersys_55Athersys 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 neurological, cardiovascular and inflammatory and immune disease. The company currently has clinical-stage programs including for the treatment of stroke, acute myocardial infarction, acute respiratory distress syndrome and other areas. 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. www.athersys.com

bluebird-bio_150x57With its lentiviral-based gene therapies, T-cell immunotherapy expertise and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer. bluebird bio’s gene therapy clinical programs include its Lenti-D product candidate for the treatment of cerebral adrenoleukodystrophy and its LentiGlobin BB305 product candidate for the treatment of transfusion-dependent β-thalassemia and severe sickle cell disease. bluebird bio’s oncology pipeline is built upon the company’s leadership in lentiviral gene delivery and T-cell engineering. bluebird bio’s lead oncology program, bb2121, is an anti-BCMA CAR-T program partnered with Celgene. bluebird bio also has discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline. www.bluebirdbio.com

CellGenix is a leading global supplier of high-quality reagents and tools for cell and gene therapy and regenerative medicine applications. As the first company to obtain a GMP manufacturing authorization for cell processing in Europe, we have more than 20 years of expertise in GMP manufacturing and development of cell therapy products. We offer a comprehensive product portfolio in combination with expert regulatory and technical support to ensure a seamless transition from research to commercialization. Our products combine a maximum of quality and safety due to the state-of-the-art production, stringent in-house quality control and comprehensive documentation. www.cellgenix.com

CTI_130_websiteCTI Clinical Trial and Consulting Services is a global, privately held, full-service contract research organization (CRO), delivering a complete spectrum of clinical trial and consulting services throughout the lifecycle of development, from concept to commercialization. CTI’s focused therapeutic approach provides pharmaceutical, biotechnology and medical device firms with clinical and disease area expertise in rare diseases, regenerative medicine/gene therapy, immunology, transplantation, nephrology, hematology/oncology, neurology, infectious diseases, hepatology, cardiopulmonary and pediatric populations. CTI also offers a fully integrated multi-specialty clinical research site that conducts Phase I-IV trials. CTI has a passion for helping life-changing therapies succeed in chronically and critically ill patient populations. With clinical trial experience across six continents, CTI partners with research sites, patients and sponsors to fulfill unmet medical needs. CTI is headquartered in Cincinnati, OH, with operations across North America, Europe, Latin America and Asia-Pacific. www.ctifacts.com

DiscGenics_140x57DiscGenics is a biotechnology company developing and commercializing advanced spinal therapeutics to treat patients with diseases of the intervertebral disc. With back pain being such a significant problem globally and a burden on the healthcare system, DiscGenics technology has the potential to increase quality of life for patients and to reduce costs to the healthcare system, while providing a significant revenue opportunity. DiscGenics is the only company to utilize allogeneic therapeutic progenitor cells derived from intervertebral disc to treat the disc. The company’s first product, Injectable Discogenic Cell Therapy (IDCT), is starting clinical testing for the treatment of moderate degenerative disc disease (DDD). www.discgenics.com

EBD_60_websiteEBD Group’s overriding mission is to help collaborations get started across the life science value chain. Our range of partnering conferences has grown to become the largest and most productive conference platform in the industry. Each one of our seven landmark events held in key life science markets around the world is powered by our state-of-the-art partnering software, partneringONE, that enables delegates to efficiently identify and engage with new opportunities via one-to-one meetings. Today our events (BIO-Europe, BIO-Europe Spring, BioPharm America, Biotech Showcase, ChinaBio Partnering Forum, Cell & Gene Exchange, and BioEquity Europe) annually attract more than 12,000 senior life science executives who engage in over 43,000 one-to-one partnering meetings. These vital one-to-one engagements are the wellspring of deals that drive innovation in our industry. www.ebdgroup.com

Fresenius-Kabi_110x57Fresenius Kabi is a leading global healthcare company that focuses on pharmaceuticals and medical devices used to care for critically and chronically ill patients. We bring over 60 years of experience advancing the fields of cell collection and separation with a deep expertise in closed-system processing. We recently launched Lovo, the only cell processing system that washes and concentrates white blood cells using filtration technology, specifically to serve the needs of the cellular therapies community. Fresenius Kabi’s U.S. headquarters are located in Lake Zurich, Illinois. www.fresenius-kabi.us/lovo

Informa_110_websiteInforma is one of the world’s leading knowledge providers. We create and deliver highly specialized information through publishing, events, training, market intelligence and expertise, providing valuable knowledge to individuals, businesses and organizations around the world. Informa provides authoritative research and analysis and up-to-the-minute business news, comment and events for all sectors of the healthcare, medical and life sciences communities. Informa Business Information (IBI) is one of the world’s leading providers of industry and drug news, analysis and data to the global pharmaceutical industry.

Kawasaki_120_websiteKawasaki is committed to providing customers unique business solutions with our innovative technologies to meet diverse societal needs worldwide. Our newly developed Automated Cell Processing System: AUTO CULTURE is intended for use in clinical research, practice and commercialization to support advances in regenerative medicine and cell therapy featuring the following capabilities: safe, stable and scalable automated production of cells; contamination control; high versatility and expandability; and reliable traceability. With our system you can reduce the initial investment in your cell processing facility as well as the running cost. Search “Kawasaki AUTO CULTURE” for more information.

LMRI_150_websiteThe Lowy Medical Research Institute (LMRI) was established in 2005, with the goal of carrying out innovative research into human diseases. LMRI is currently focused on the study of a neuro-/vasculodegenerative disease of the retina, idiopathic juxtafoveal macular telangiectasia type 2, or MacTel. In addition to the Institute with a full time staff in La Jolla, California, LMRI supports multinational natural history and interventional clinical trials as well as international laboratory collaborations in North America, Europe, Asia and Australia. www.lmri.net

MEDIPOST_72x57MEDIPOST’s research and development is focused on novel off-the-shelf allogeneic stem cell therapeutics using umbilical cord blood-derived stem cells in the disease areas of Osteoarthritis (OA), Broncho-pulmonary Dysplasia (BPD) and Alzheimer’s disease (AD). MEDIPOST’s flagship product, CARTISTEM® for OA was approved by the regulatory agency in Korea as an allogeneic stem cell product in 2012 and to date, over 3,500 patients have been treated on the market with an excellent long-term safety and efficacy profile. CARTISTEM® has also successfully completed a Phase I/IIa trial in the U.S. PNEUMOSTEM® for the prevention of BPD in premature infants, completed Phase I and Phase II clinical trials in Korea, while currently undergoing a Phase I/II clinical trial in the U.S. PNEUMOSTEM® has received Orphan Drug Designation in Korea, the U.S. and EU. NEUROSTEM® for AD is in a Phase IIa clinical trial in Korea after successfully completing the first-in-human Phase I trial in Korea with patients suffering from AD. www.medipostamerica.com | www.medi-post.com

Nordmark Biochemicals offers translational collagenases and other proteases used for tissue dissociation and cell isolation. The Collagenase NB products manufactured by Nordmark were previously marketed and distributed worldwide by SERVA. In 2018, Nordmark took over distribution in order to offer production, sales, marketing and support from one source. Our product portfolio includes Collagenase NB 6 GMP Grade for isolation of a variety of cells destined for clinical applications, and animal-free Collagenase AF-1 GMP Grade and Neutral Protease AF GMP Grade, the world’s first animal-free GMP grade products. Our pharmaceutical manufacturing standards result in high lot-to-lot consistency, safety and superior quality to guarantee cell viability and high yields. Supporting documentation and access to Drug Master Files are available for GMP grade users. More affordable research grade enzymes can be used for protocol development before transitioning to the GMP grades with similar enzymatic activities for an easy transition to the clinic. www.nordmark-biochemicals.com

Precision_130Precision BioSciences is a privately held biotechnology company dedicated to improving lives through its next generation gene editing technology, ARCUS. Precision’s approach is based on a proprietary synthetic enzyme, the ARC endonuclease, which features coordinated target site binding and DNA cutting to ensure precise editing outcomes. Uniquely small and monomeric, ARC nucleases are readily delivered to target sites throughout the genome and across cell types and tissues. The value of the ARCUS editing platform is enhanced by Precision’s extensive patent portfolio, established freedom to operate and deep collaborative relationships, positioning Precision to create products that solve significant problems in oncology, genetic disease, agriculture and beyond. Our team includes pioneers in genome engineering and a staff of experienced, committed Precisioneers. Working together in the vibrant innovation center of Durham, NC, we are excited to lead the next wave of medical and scientific possibilities through continuous gene editing innovation. www.precisionbiosciences.com

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO’s NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology licensees that include Adverum, Audentes, AveXis, Biogen, Dimension, Esteve, Lysogene, and Shire are applying the NAV Technology Platform in the development of a broad pipeline of product candidates in multiple therapeutic areas. REGENXBIO is currently developing its own gene therapy product candidates in the retinal, metabolic and neurodegenerative therapeutic areas. Our gene therapy product candidates are designed to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins that are intended to impact disease. Through a single administration, our gene therapy product candidates are designed to provide long-lasting effects, potentially significantly altering the course of disease and delivering improved patient outcomes. www.regenxbio.com

A critical factor facing the industry is choosing technologies and partners that can deliver scale. The challenges inherent in translating early clinical trials to a reproducible and robust process suitable for scalable commercial production are very significant. Each personalized drug product ultimately depends on a sustainable and consistent supply chain with lots of moving parts. Configured to your exact demands, using an industry-standard framework, TrakCel’s comprehensive supply chain platform ensures every resource is managed, monitored, recorded and is ultimately performed exactly as required. From sample collection through logistics, manufacturing and final transplantation, with comprehensive chain of custody, chain of identity and traceability, TrakCel’s proven technology integrates and orchestrates every aspect of your autologous and allogeneic supply chains in a scalable, compliant and validated technology platform. www.trakcel.com

Universal Cells is developing stem cell therapies that overcome immune rejection and can be used to create truly off-the-shelf stem cell-based therapeutic products. Using the company’s proprietary genome editing approach, Universal Cells engineers stem cells to prevent expression of polymorphic human leukocyte antigen (HLA) molecules, removing the mechanism by which donor tissue rejection occurs and creating Universal Donor Stem Cells that are compatible with every patient. The company has established major strategic partnerships to help advance its Universal Donor Cell Program to the clinic. By combining the safest gene editing approach with a deep understanding of stem cell immunology, the company is positioned to play a central role in the commercialization of pluripotent cell-based therapies. www.universalcells.com

Voyager_110Voyager Therapeutics is a clinical-stage gene therapy company developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV (adeno-associated virus) gene therapy through innovation and investment in vector engineering and optimization, manufacturing and dosing and delivery techniques. The company’s pipeline is focused on severe neurological diseases in need of effective new therapies, including advanced Parkinson’s disease, a monogenic form of amyotrophic lateral sclerosis (ALS), Huntington’s disease, Friedreich’s ataxia, frontotemporal dementia and Alzheimer’s disease and severe, chronic pain. Voyager has broad strategic collaborations with Sanofi Genzyme, the specialty care global business unit of Sanofi, and the University of Massachusetts Medical School. Founded by scientific and clinical leaders in the fields of AAV gene therapy, expressed RNA interference and neuroscience, Voyager Therapeutics is headquartered in Cambridge, MA. www.voyagertherapeutics.com

The Advanced Therapies business unit of WuXi AppTec is a Contract Development and Manufacturing Organization (CDMO) that reduces the complexities of the high-touch, multi-vendor biopharmaceutical production model by providing platforms that enable cell and gene therapies to be developed, manufactured and released faster and with greater predictability. Since 2004, WuXi AppTec has supported Advanced Therapy manufacturing programs from their Philadelphia, PA campus, which today includes over 220,000 square feet of GMP compliant manufacturing space. www.advancedtherapies.com