Holland Innovation Network Team Boston: Walter de Wit Julia Knoeff

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1 Holland Innovation Network Team Boston: Walter de Wit Julia Knoeff Boston and Cambridge in Massachusetts are innovation-focused hubs with world-leading universities such as Harvard, Boston University and MIT. This creates an ecosystem for cutting-edge research in the field of regenerative medicine. For example, in June 2015 investigators at Massachusetts General Hospital reported building bio artificial organs to engineer the forelimbs of rats. Additionally they provided evidence that the same approach could be applied to the limbs of primates. 1 Within the field of regenerative medicine specifically, Boston and Cambridge host groundbreaking research institutes such as the Harvard Wyss Institute, the Harvard Stem Cell Institute, the MIT Whitehead institute and the Boston University Center for Regenerative Medicine. These research institutes cooperate with well-known hospitals such as Brigham and Women s Hospital, Children s Hospital and Massachusetts General Hospital. From diabetes, liver disease, cancer and heart disease, the regenerative medicine possibilities are endless with medical devices, artificial organs, tissue engineering, biomaterials and cellular therapies. This overview gives insight into the research, companies and events in the Northeast of the US on regenerative medicine in Research The top-notch hospitals and academic institutions provide Massachusetts with a plethora of worldrenowned specialized research institutes in regenerative medicine. Each of the research institutions differentiates itself from others by following a specific research direction. Harvard s Wyss Institute 2 for example looks to revolutionize healthcare by creating biologically inspired materials, devices and disease reprogramming technologies that imitate how living tissues and organs self-organize and naturally regulate themselves. MIT s Whitehead Institute 3 is a world-renowned non-profit research institution dedicated to improving human health through basic biomedical research. The Center for Regenerative Medicine (CReM) 4 is a collaborative effort among scientists at Boston University and the Boston Medical Center that aims to advance stem cell research and regenerative medicine for the sake of patients. The research of the CReM is particularly focused on those suffering from diseases commonly seen at the Boston Medical Center. This practical and specialized way of innovating also shows in the research institutions of hospitals in Massachusetts. For example, The Brigham and Women s Hospital s Regenerative Medicine Center 5 aims to integrate the talents of engineers, physicists and mathematicians with those of biologists and clinicians. This combination of stem cell biology with engineering and nanotechnology makes the BWH Regenerative Medicine Center unique. Furthermore, there are institutes specialized in specific areas of the body. For example, the Massachusetts Eye and Ear s Ocular Regenerative Medicine Institute (ORMI) 6, a Harvard Medical School teaching hospital, believe that tissue and stem cell therapies hold enormous promise for repairing ocular tissues. Investigators in the Ocular Regenerative Medicine Institute lead the effort to develop revolutionary methods to regenerate eye tissues for various diseases, including age-related macular degeneration (AMD), glaucoma, corneal disease, and ocular cancer. Notable is the cooperation between the different institutions in Massachusetts, made possible by their proximity in Boston and Cambridge. The Harvard Stem Cell Institute (HSCI) 7 was a pioneer in advancing the understanding of the potential of stem cell biology. Now the institute Pagina 1 van 5

2 focuses on getting stem cell-based treatments to patients as quickly as possible, aimed at treatments for diseases of aging, the blood, metabolism, and neurological disorders. HSCI is crucial to the cooperative eco-system as they bring more than 1,000 scientists together from the schools and affiliated hospitals of Harvard, as well as implement new collaborative academic and industrial models. For example, Massachusetts General Hospital Research Institute 8 Center for Regenerative Medicine is a part of the HSCI. They are dedicated to understanding how tissues are formed and may be repaired. Additionally, the world-famous Ott Laboratory for Organ Engineering and Regeneration 9, is linked to the Harvard Stem Cell Institute, MIT, Harvard Medical School, the New England Organ Bank, and the clinical departments of MGH. Organizations Large and small companies are leading revolutions in different fields of regenerative medicine in Massachusetts. From cell therapy, innovative treatment in the orthopedic space, gene therapy to organ-on-a-chip technology. GE Healthcare s 10 $4.0 billion life sciences business aims to accelerate innovation in molecular and precision medicine by providing expertise, tools and solutions for a wide range of applications. This includes basic research of cells and proteins, drug discovery, cell therapy and regenerative medicine. Histogenics 11 is a regenerative medicine company focused on developing and commercializing products in the musculoskeletal segment of the marketplace. Their regenerative medicine platform provides solutions that can be utilized individually or in concert to treat musculoskeletal-related conditions. Their first product candidate, NeoCart, leverages their platform to provide an innovative treatment in the orthopedic space, specifically cartilage damage in the knee. Novare Pharmaceuticals 12, based in Boston develops therapeutic products that have the potential to reduce inflammation from disease or traumatic injury, minimize breast cancer tumor growth and metastasis, prevent collateral fibrosis from radiation therapy, and help the body regenerate fat tissue. Bluebird Bio 13 is leading the gene therapy revolution. Their product platforms encompass gene therapy, cancer immunotherapy and gene editing with the potential to treat and possibly cure, a broad range of serious diseases. Voyager Therapeutics 14 is a clinical-stage gene therapy company developing life-changing treatments for severe diseases of the central nervous system. Other successful regenerative medicine companies including many with a focus on stem cell therapy and gene therapy are AuxoCell Laboratories, Inc. 15, Asymmetrex 16, Celyad 17, InformGenomics 18, Vor Biopharma 19, AvroBio 20, Pathfinder 21, Astellas Institute for Regenerative Medicine (AIRM), 22 and BioAxone 23. In the relatively new field of organ-on-a-chip technology, Emulate 24 is a fast-growing Boston start-up that emerged out of the Harvard Wyss Institute. Based on organs-on-chips technology Emulate has developed an integrated system that provides a window into the inner-workings of the human body. They can predict human response with greater precision and control than today s cell culture or animal-based testing methods. Another recently emerged company is ORIG3N 25, founded only in 2014, is a regenerative medical company that has crowdsourced blood samples to create the world s biggest and most diverse bio-repository. ORIG3N has already raised over $15 million, with a series A in December 2015 of $12.5 million. Lastly, the first-of-its-kind shared laboratory space LabCentral in Cambridge, provides space for high-potential life-sciences and biotech startups. The 28,000 square-foot facility is located in the heart of the Kendall Square, Cambridge, biotech innovation hub, LabCentral is a first-of-its-kind Pagina 2 van 5

3 shared laboratory space designed as a Launchpad for high-potential life-sciences and biotech startups. 26 Opportunities Massachusetts Life Sciences funding 2015 The Massachusetts Life Sciences Center (MLSC) announced in 2015 more than $17 million in funding for life sciences-related capital projects. 27 Boston University and Boston Medical Center were awarded $1,740,000 to launch an expansive Lung Regeneration Initiative (LRI) as part of their Center for Regenerative Medicine. January, 2016 MIT researchers taking steps forward in organ-on-a-chip technology 28 In a step toward personalized drug testing, researchers coax human stem cells to form complex tissues. The researchers now hope to investigate whether they can use the technique to grow other organs on demand, such as a pancreas. March Investors ready to invest in organ-on-a-chip technology The Cambridge, MA-based startup Emulate raised $40 million in equity funding since it was spun out of Harvard University s Wyss Institute in The company now has several partners including pharmaceutical companies Merck and Johnson & Johnson. 29 July Harvard Business School 30 The Harvard Stem Cell Institute organized a course at Harvard Business School for The Business of Regenerative Medicine How to Build a Company. This 2.5-day course focused on the critical issues associated with conceptualizing, developing, and building a company in the field of regenerative medicine. August, Converting skin cells to donor organs for animals 31 Scientists at Massachusetts General Hospital, specifically the Ott laboratory for organ engineering and regeneration, successfully converted skin cells to donor organs for animals. This is done by stripping away the muscle cells in the heart, leaving the rest of the structures intact, and then rebuilding the heart with new muscle cells. It could provide people with patches that replace damaged tissue, and save heart transplant patients from rejecting their new organs. Events 7-8 July 2016 Select Bio s organ on a chip world congress 32 The Second Annual Organ-on-a-Chip World Congress 2016 focuses on an important and expanding field wherein cells are being assembled using microfluidics and Lab-on-a-Chip technologies in a functionally-relevant manner. These clusters of "assembled cells" have functional significance and can mimic in vivo organ structure. September th 2016 Partnerships in Regenerative Medicine and Advanced Therapies during the 9 th annual international partnering conference BioPharm America BioPharm America is where biotech industry partnerships get started. Equipped with partneringone, the world s leading web-based partnering system for the life science industry, Pagina 3 van 5

4 BioPharm America is the only event in North America based on the same reputable formula as EBD Group s acclaimed European events BIO-Europe and BIO-Europe Spring. September th 2016, from Stem Cells to Human Development, Southbridge, MA Following on from a highly successful inaugural meeting in 2014, the second in this series of meetings From Stem Cells to Human Development brings together scientists with a common interest in understanding human development using stem cell systems. September th 2016, HubWeek s Horizons in Regenerative medicine, Cambridge, MA This is hosted by HubWeek in partnership with the Federal Reserve Bank at Harvard University in Cambridge. Over the course of a few days, there is a large focus on regenerative medicine with three specific sessions. One of the sessions is A quantum leap in Diabetes Treatment, about finding a cure for diabetes. Another focuses on organs-on-chips, diseases in dishes and the potential impact of these studies. Lastly, Ageless Aging focuses on prospects for slowing down, and even reversing, some of the effects of aging. April 26, April 27, 2017, Global Technology Community 34, Boston, MA The 14 th Stem Cell Research & Regenerative Medicine Conference in Boston. This conference presents information regarding cutting-edge developments in all areas of stem cell research. Topics of discussion include recent developments in pre-clinical and clinical trials of stem cell therapy, regenerative medicine and tissue engineering, cancer stem cells, immunotherapy, stem cell reprogramming, and regulatory policies regarding stem cell research. Finances The Alliance for regenerative medicine (ARM) is a global advocate for regenerative and advanced therapies and provides reports on the regenerative medicine industry. 135 Their 2015 ARM annual report shows that companies involved in regenerative medicine continue to sustain elevated interest from investors of all types into The report shows regenerative medicine total financings as $10.8 billion, which is up 106% compared to This was divided mostly into cell therapy with $7.0 billion, gene and gene-modified cell therapy with $6.8 billion and tissue engineering with $806.8 million. The financing types range and 2015 shows more IPO s, follow-ons, corporate partnerships and venture capital financing than This shows a high increase in investments in the field of regenerative medicine Pagina 4 van 5

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