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Technical Inisights: Advances In Stem Cell Research
 

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    Technical Inisights: Advances In Stem Cell Research Technical Inisights: Advances In Stem Cell Research Presentation Transcript

    • Advances in Stem Cell Research D2AD – TI HC February 2011
    • Table of Contents
      • Executive Summary
        • Scope
        • Research Methodology
      • Key Findings
      • Sectoral Analysis
      • Technology Snapshot
        • Stem Cells--Current Technology Trends
        • Stem Cell Technology Roadmap
        • Government Grants and Stimulus Funding
        • GAP Analysis--Stem Cell Developer Community and Need for Funding
      • Assessment of Investment Ecosystems
        • Investor Networks and Recent Investment Climate
        • Venture Capital/Private Equity
        • VC Divisions of Corporates
        • Governmental Policies in Favor of Incubators
        • Technology Incubators
        • Stem Cell Policies
        • Funding Patterns and Investor Inclination
        • Future Plans And Expectations Of Investors
      • Analyst Insight and Recommendations
        • Assessment of Funding Sources
        • Investment Prospects and Opportunity Evaluation for Investors
        • Smart Scouting and Procurement Strategies for Stem Cell Companies
      • Key Patents
      • Contacts
    • Technology Snapshot Stem cells have the potential to develop into many different cell types in the body during early life and growth. They also serve as an internal repair system, dividing to replenish other cells. Distinguished from other cells by virtue of being unspecialized cells capable of Renewing themselves through cell division and that under certain physiologic conditions they can be induced to become tissue or organ-specific cells with particular functions. Types: Embryonic: Derived from embryos, that, in turn, are mostly derived from eggs that are fertilized in vitro and donated for research with donor’s permission. Adult Stem Cell: Undifferentiated cell found among differentiated cells in tissue/organ. Can differentiate itself to yield some or all of the major specialized cell types of that tissue/organ. iPSC: Adult cells genetically reprogrammed to an embryonic stem-cell like state and forced to express genes and factors to define the properties of embryonic stem cells.
      • Applications:
      • The use of stem cells has augured a new field called regenerative
      • medicine or cell therapy—replacing damaged or diseased cells in
      • body with new ones. The applications of stem cells can basically be
      • classified into the following broad categories:
      • Development of therapies and treatments in the following diseases:
      • Juvenile Type I diabetes
      • Nervous system diseases
      • Primary Immunodeficiency diseases
      • Bone and Cartilage diseases
      • Cancer
      • Research in the following areas:
      • Developmental Biology
      • Models of human disease constrained by current animal
      • and cell culture models.
      • Transplantation
      • Gene Therapy
      • The major factors influencing these applications are:
      • Stem Cell policies
      • Public funding resources for basic research
      • Private funding sources for application-stage research
      • Some Major Companies:
      • Advanced Cell Technologies
      • Geron Corporation
      • iPierian
      • Cytori Therapeutics
      • Osiris Therapeutics
      • Mesoblast
      • Aastrom
      • A majority of the companies (~70%)
      • are based out of the United States.
      • The Stem Cell Summit is an annual event
      • held in the United States to provide opportunities
      • for industrial and academic leaders in this field to
      • interact.
      Stem cells have the potential to develop into many different cell types in the body during early life and growth. They also serve as an internal repair system, dividing to replenish other cells. Distinguished from other cells by virtue of being unspecialized cells capable of Renewing themselves through cell division and that under certain physiologic conditions they can be induced to become tissue or organ-specific cells with particular functions. Types: Embryonic: Derived from embryos, that, in turn, are mostly derived from eggs that are fertilized in vitro and donated for research with donor’s permission. Adult Stem Cell: Undifferentiated cell found among differentiated cells in tissue/organ. Can differentiate itself to yield some or all of the major specialized cell types of that tissue/organ. iPSC: Adult cells genetically reprogrammed to an embryonic stem-cell like state and forced to express genes and factors to define the properties of embryonic stem cells.
      • Applications:
      • The use of stem cells has augured a new field called regenerative
      • medicine or cell therapy—replacing damaged or diseased cells in
      • body with new ones. The applications of stem cells can basically be
      • classified into the following broad categories:
      • Development of therapies and treatments in the following diseases:
      • Juvenile Type I diabetes
      • Nervous system diseases
      • Primary Immunodeficiency diseases
      • Bone and Cartilage diseases
      • Cancer
      • Research in the following areas:
      • Developmental Biology
      • Models of human disease constrained by current animal
      • and cell culture models.
      • Transplantation
      • Gene Therapy
      • The major factors influencing these applications are:
      • Stem Cell policies
      • Public funding resources for basic research
      • Private funding sources for application-stage research
      Stem cells have the potential to develop into many different cell types in the body during early life and growth. They also serve as an internal repair system, dividing to replenish other cells. Distinguished from other cells by virtue of being unspecialized cells capable of Renewing themselves through cell division and that under certain physiologic conditions they can be induced to become tissue or organ-specific cells with particular functions. Types: Embryonic: Derived from embryos, that, in turn, are mostly derived from eggs that are fertilized in vitro and donated for research with donor’s permission. Adult Stem Cell: Undifferentiated cell found among differentiated cells in tissue/organ. Can differentiate itself to yield some or all of the major specialized cell types of that tissue/organ. iPSC: Adult cells genetically reprogrammed to an embryonic stem-cell like state and forced to express genes and factors to define the properties of embryonic stem cells.
      • Some Major Companies
      • Advanced Cell Technologies
      • Geron Corporation
      • iPierian
      • Cytori Therapeutics
      • Osiris Therapeutics
      • Mesoblast
      • Aastrom
      • A majority of the companies (~70%)
      • are based out of the US.
      • The Stem Cell Summit is an annual event
      • held in the US to provide opportunities
      • for industrial and academic leaders in this field to
      • interact.
      • Applications
      • The use of stem cells has augured a new field called regenerative
      • medicine or cell therapy--replacing damaged or diseased cells in
      • body with new ones. The applications of stem cells can basically be
      • classified into the following broad categories:
      • Development of therapies and treatments in the following diseases:
      • Juvenile Type I diabetes
      • Nervous system diseases
      • Primary immunodeficiency diseases
      • Bone and cartilage diseases
      • Cancer
      • Research in the following areas:
      • Developmental biology
      • Models of human disease constrained by current animal
      • and cell culture models.
      • Transplantation
      • Gene therapy
      • The major factors influencing these applications are:
      • Stem cell policies
      • Public funding resources for basic research
      • Private funding sources for application-stage research
      Stem cells have the potential to develop into many different cell types in the body during early life and growth. They also serve as an internal repair system, dividing to replenish other cells. Distinguished from other cells by virtue of being unspecialized cells capable of renewing themselves through cell division and that under certain physiologic conditions they can be induced to become tissue or organ-specific cells with particular functions. Types Embryonic: Derived from embryos, that, in turn, are mostly derived from eggs that are fertilized in vitro and donated for research with donor’s permission. Adult Stem Cell: Undifferentiated cell found among differentiated cells in tissue/organ. Can differentiate itself to yield some or all of the major specialized cell types of that tissue/organ. iPSC: Adult cells genetically reprogrammed to an embryonic stem-cell like state and forced to express genes and factors to define the properties of embryonic stem cells. Source: Frost and Sullivan
    • 2012 2013 2014 2011 2015 Stem Cells--Technology Roadmap 2016 2017 2018 2019 2020 2021 Advent of iPS cell -based therapies as Alternatives to hES lines. Use of APSCs to develop therapies for cardiovascular and musculoskeletal diseases Development of standardized IP system for protecting iPSCs. Co-development of hES therapies. Exclusive use of stem cells for drug screening, eliminating animal testing altogether. Extensive use of iPSCs to develop cancer therapies (NA). Use of cord blood and hES cells for therapeutic development (APAC). Use of iPSCs for gene therapy, drug screening, and also general toxin screening Well-developed therapies using APSCs for diseases , such as diabetes and myocardial infarction. Refinement of iPSC differentiation efficiency and research tools for genetic programming (iPSC production) Source: NIH, EuroStemCell, Nature Biotech
    • Governmental Grants and Stimulus Funding—USA
      • CALIFORNIA
      • Upcoming companies: Cytori Therapeutics, iPerian,
      • StemGent, NeuroGeneration, and California Stem Cell.
      • APSC and hESC (Geron Corp.) presence
      • about 3 of the 4 major iPSC companies here.
      • CIRM --$300 million every year
      • NIH --$3.15 billion in research grants
      • Major venture corporations--Kleiner, Perkins, Caufield
      • and Byers, Google Ventures, Morganthaler
      • NEW YORK
      • One of the last states to approve state funding
      • Empire State Stem Cell Trust--$121 million till 2009, $600
      • million pledged over 11 years
      • Notable companies --BrainStorm Cell Therapeutics,
      • NeoStem.
      • Grant concentration on research institutions—Albert Einstein,
      • Sloan-Kettering, Rochester , and Columbia
      • MARYLAND
      • Established and strong state funding--Maryland Stem Cell
      • Research Fund (MSCRF), in collaboration with TEDCO (Maryland
      • Technology Development Corporation)
      • $54 million awarded in state grants so far
      • Notable companies--GlobalStem, Osiris Therapeutics, NeuralStem,
      • and Vesta Therapeutics
      • MASSACHUSETTS
      • No established stem cell funding initiative.
      • General funding proposal from
      • Massachusetts Life Science Initiative.
      • $45 million in grants
      • National Stem Cell Bank and first hESC
      • registry to be created.
      • MPM Capital and Healthcare Ventures
      • housed here.
      • Notable companies--Advanced Cell
      • Technologies and ViaCell
      • NEW JERSEY
      • First state to finance hESC research including prohibited lines
      • New Jersey Stem Cell Initiative -- $15 million in funds so far.
      • Notable companies—Progenitor Cell Therapies (CMO) and
      • Celgene Cellular Therapeutics.
      • Funding was also put on hold in 2008 for the New Jersey Stem
      • Cell Center
      Source: EDGAR, Primaries, CIRM database
    • Governmental Grants and Stimulus Funding—Europe
      • UK
      • Notable national bodies: UK Stem Cell Initiative, Cambridge
      • Stem Cell Initiative
      • Funding bodies: Medical Research Council, Biotechnology,
      • and Biological Sciences Research Council, Wellcome Trust
      • Lack of venture funding, especially for technology that is
      • feasible, but needs second or third round of funding ( £1- £ 5m)
      • Notable companies: ReNeuron, Capsant, Reinnervate (applied)
      • and EpiStem (CRO)
      • General consensus: Flourishing stem cell industry difficult despite
      • permissive policy due to lack of venture funding.
      • SWEDEN
      • Funding bodies: Swedish Research Council,
      • Foundation for Strategic Research, Wallenberg Foundation.
      • Commercialization of research is relatively easier
      • due to availability of funds from members of the
      • Swedish Venture Capital Association.
      • Active collaboration with US companies and seeking
      • collaborations in China, Australia, Singapore, and India.
      • Government and private interest in stem cells, two
      • major research centers (Karolinska and Lund) and
      • permissive policy.
      • Interest from foreign venture firms, such as ABN Amro and
      • MPM capital.
      • Strong research partnerships with US firms; actively
      • seeking collaborations in China, Singapore, Australia, and India.
      • DENMARK
      • Major government funding sources: Danish Research
      • Council (DASADOC), Danish National Research Foundation,
      • Danish Council for Strategic Research, and Danish National
      • Advanced Technology Foundation.
      • Corporate venture funding: Roche and Novo Nordisk.
      • Government initiatives for stem cell research: DanStem at the
      • University of Copenhagen, Danish Centre for Stem Cell
      • Research.
      • Notable company: NsGene A/S
      Source: EuroStemCell, Primaries
    • Governmental Grants and Stimulus Funding--APAC AUSTRALIA The most active participant in the stem cell sector from both an academic and an industry point of view. Australian Stem Cell Center (ASCC)—comprehensive stem cell organization with a strong focus on partnering and networking. Active incubator support and commercialization policies from the University of Queensland, University of Western Australia, ASCC And the Monash Institute of Medical Research (MIMR). Notable absence of work in the iPSC domain—critical at this stage. Notable companies: Mesoblast, Nephrogenix, Stem Cell Sciences Top research institutes: University of Western Australia, MIMR, and Peter McCallum Cancer Center.
      • SINGAPORE
      • Initiatives since 2004 from major funding sources such as the Biomedical
      • Research Council (BMRC) and SSCC (Singapore Stem Cell Consortium).
      • Work on iPSC cells from the Genomic Institute of Singapore.
      • Government and private funding for a developing stem cell industry initiative.
      • Notable names: ES Cell International and Stem Cell Technologies Inc.
      • Major academic involvement for fundamental research: A*Star Biomedical
      • Sciences Research Institutes, National University of Singapore and Nanyang
      • Technical University
      • Permissive stem cell research policies (human cloning is forbidden).
      • SOUTH KOREA
      • Permissive stem cell policies
      • allowing for both human and
      • therapeutic cloning.
      • CHA Biotech, the premier stem
      • cell research facility.
      • Collaborations with leading US
      • stem cell companies, such as
      • Advanced Cell Technology.
      • Federal funding an issue due to
      • 2004-05 scandal with stem cell
      • cloning
      • ISRAEL
      • Second highest publication of stem cell research per capita globally.
      • 10 startups in the stem cell industry focusing on adult and embryonic
      • stem cell research and applications.
      • Challenge—no established system of stem cell funding or separate
      • government-allocated funds for stem cell research. Only non-private
      • sources of funding are the Ministry of Health and Trade Stem Cell
      • Consortium.
      • Renowned institutes and researchers in both adult and embryonic cells
      • due to permissive policies: Weizman Institute, Tel Aviv University,
      • Technion Institute.
      • Extensive collaborations with USA federal, private, and corporate funds.
      • Notable companies: Gamida, PluriStem, and CellCure Neurosciences
      Source: ASCC, UKSCF, Frost and Sullivan
    • Stem Cell Policies (Global) Permissive Flexible Restrictive
    • Funding Patterns and Investor Inclinations
      • KEY FACTS
      • Almost 70% of the top players in the global stem cell industry are based on APSC research and APSC-derived therapies. Embryonic stem cells occupy around 17% of the top 21 list, around a fourth of the APSC share. ‘Others’ denote stem cell application technologies and cord blood cell banks.
      • Maximum global funding is also concentrated in this area of stem cell research
      • Embryonic stem cells are also less in share due to policy restrictions depending on geography. Many European countries , such as France and Switzerland that have otherwise robust IP systems have been unable to capitalize on this sector due to the absence of permissive policies.
      • iPSCs currently have a very small share in the top 21. The technology for producing iPSCs is still very nascent and does not have much presence outside the US at present.
      Stem cell funding depends on a number of fators that should be optimal for maximum funding Policy Individual country policies play a very important part in stem cell funding. hESC research has restrictions in many developed countries , such as France and Germany, rendering this industry weak there. Company Stage The developmental stage of the company is also a decision-making factor, in particular for venture capitalists (VC). VCs tend to fund middle to late stage companies while federal or state funding is more available for early stage ventures. Time to Market Time to market is the most important facture for VC-backed funding. VCs typically extend short-term financing rounds , and therefore, look for quick return on investment. This can only be achieved by a short time to market. Country Initiatives Countries, such as China, Singapore, Australia, and Sweden have taken extensive initiatives to lure researchers, build incubation centers, and set up local as well as commercialization vehicles for their stem cell sector. Collaboration and tech transfer A strong academia-industry and international collaboration structure is necessary for translating academic research to a viable treatment or product. This is especially visible in countries, such as Australia, US, and Sweden. Economy The economic crisis of 2008-09 has a put a significant strain on US funding in stem cells from the VC sector. Federal funding has not suffered as much , but higher federal funding implies taxpayer bonds that have to be paid back at stipulated times. FACTORS INFLUENCING STEM CELL FUNDING Source: Frost and Sullivan
    • Funding Patterns and Investor Inclinations--Contd. North America Europe APAC Early-stage Middle Stage Late Stage Economic crisis of 2008-09—tightening of VC funds for early stage as time to market is high Other than Geron, most of these are APSC-based. Late stage companies exclusively IPO. Europe does not have any late stage companies. Venture system is strong in countries, such as like Sweden and Denmark. Early-stage companies also qualify for stem cell funding. hESC—still mostly research. APAC has late stage companies due to Israel and Australia. Other than IPO, another big funding source is international federal and VC funds. Government Funding Venture capital/corporate venture funding IPO/institutional investors Federal funding is high for early-stage companies in China and Singapore. However, Australia is undergoing a slow-down. The above grid provides an overview of the investing patterns according to the stage that the stem cell company is in and the geographical region where it is. The three regions shown on the X-Axis are the broad stem cell ‘hotpsots’. Funding has been classified in terms of federal, private/VC/corporate and public/institutional. The yellow boxes represent examples of companies best fitting the axial characteristics of the rectangle. Stemina, StemGent, PrimeGen Capsant, Stemedica Intl., Beike Biotech, Stem Cell Sciences Pty. Geron, Osiris Cytori, VistaGen NeuroNova, NsGene S*Bio, CellCure, ProChon ES Cell Intl., Abcam, Gamida
    • Investment Prospects and Opportunity Evaluation for Investors Stem Cells Nephrogenix, Australia: Researchers at Nephrogenix investigate both embryonic and adult stem cells for use in cellular therapy for kidney diseases. It has long been assumed that kidney development ceased at birth with no prospect of regeneration of new functional units. Researchers at Nephrogenix are developing technologies to enable the differentiation of mesenchymal stem cells , such that they adopt a renal progenitor fate. It is envisaged that glomerular and tubulo-interstitial damage in the ESRD kidney can be repaired by the administration of embryonic stem cell-derived or patient-derived renal progenitor cells. The company is currently funded by the Australian Stem Cell Center and the Australian Medical Research Council. As the ASCC closed in 2010, the company is now actively looking for investors and collaborators Stemina Biomarker Discovery, WI, US: Stemina Biomarker Discovery, based out of Madison, Wisconsin, is one of the few companies to come up with DevTOXTM, a drug toxicity screening assay using stem cells as the base and metabolomics as the screening method. The assay is entirely human-based unlike previous toxicity screening models that have been rodent-based. Stemina first established a predictive model of developmental toxicity to use a tool for this assay. They then tested the model’s ability to predict teratogens by applying it against 9 different drugs. The results were 89% accurate, 80% specific , and 100% sensitive. Wisconsin has no established source of state funding and hence, the company is open to funding from private/venture/corporate investors. Stem Cell Technologies, SCTi, Singapore: This company conducts both basic and application-based research on all types of adult mesenchymal stem cells for the discovery of therapies for cardiovascular diseases, diabetes, congenital birth defects , and retinal diseases. This consortium is funded by the FDA and A*Star Institute for Biomedical Sciences. However, some venture funding and incubator support will help it translate its therapies from the lab to the ‘bedside’. Capsant, UK : High-throughput in vitro tissue platform for drug discovery using progenitor stem cells. However, as the company is based out of the UK, it is highly probable that second, third and fourth series financing rounds cannot be sourced, due to the lack of venture funds. Short-Term Long-Term Medium-Term