Stem Cells Sweden

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Stem Cells Sweden
A key area for stem cell research and investment
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A major new healthcare sector

• Stem cell research with
• potential to transform disease treatment, reduce
  costs to society, and enhance quality of life for
  millions of patients.
• scope for significant business opportunities,
  with new directions for industry innovation and
  product development.
• A unique combination of scientific know-how and
  commercialization capabilities is enabling Sweden
  to become a world force in the field.

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Business opportunities in Sweden

• Leading stem cell researchers and research
  institutes.
• Global position in culturing embryonic stem cell
  lines.
• Favorable political and ethical climate.
• Relatively easy to capitalize on innovations
  teachers exemption and technology transfer
  units
• Sophisticated and specialized domestic venture
  capital industry.

Sweden has emerged as a key area for stem cell
research and investment. Elliott Davis, General
Partner, Next Wave Funds
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Current market potential

• Licensing of stem cell lines.
• Proliferation and characterization of specialized
  lines.
• Commercialization of media that direct cell
  differentiation.
• Diagnostic methods and techniques
• Therapy development.

A number of Swedish companies have been founded
to capture business opportunities in stem cell
research.
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Good reasons to choose Sweden for stem
cell-based RD

• Source of breakthrough research achievements
• Holds a large share of human embryonic stem cell
  lines eligible for U.S. public funding.
• The legislative environment supports all areas of
  stem cell research.
• Has more than 30 stem cell research groups and
  300 people at nine Swedish institutions involved.
• Stem cell clusters are emerging at three
  prominent life sciences sites.
• Capability to cover whole value chain for
  commercialization of stem cell research.

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Stem cells a definition

• Undifferentiated cells that give rise to all of
  the bodys cells and organs. Three sources of
  stem cells embryonic, adult and fetal. Research
  performed on humans and in animal models.
• Capacity for self-renewal through repeated cell
  division, every stem cell can form two identical
  copies of itself
•   and multipotency stem cells can form progeny
  that can differentiate, i.e. develop into one of
  the different types of cells that comprise the
  living organism
• In principle, each one of the human bodys 200
  different cell types can be cultured from a
  single immature stem cell.
• Research performed on different tissue types
  Hematopoietic (blood) Neural (brain)
  Mesenchymal (connective tissue, muscles, blood
  vessels)

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From stem cell to full-fledged body cell
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Potential to cure a variety of diseases

• Blood cells Cancer, immunodeficiencies, inherited
  blood, diseases, leukaemia
• Bone cells Osteoporosis
• Cartilage cells Osteoarthritis
• Heart muscle cells Heart attacks, congestive
  heart failure
• Insulin-producing cells Diabetes
• Liver cells Hepatitis, cirrhosis
• Nerve cells Stroke, Parkinsons disease,
  Alzheimers disease, spinal cord injury, multiple
  sclerosis
• Retinal cells Macular degeneration
• Skeletal muscle cells Muscular dystrophy
• Skin cells Skin cells burns, wound healing

• Two therapeutic approaches have been
  distinguished
• Cellular therapy (i.e. cell transplantation)
• Pharmaceutical approach (signaling substances)

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High quality of research (1)
Source Boston Consulting Group, Swedish Brain
Power, 2001
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High quality of research (2)
The graph shows individual countries share of
publications in scientific journals per capita.
Publications in 24 scientific journals were
studied and classified according to each
journals impact factor.
Source Boston Consulting Group, Swedish Brain
Power, 2001
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Large commercial potential

• High-potential diagnoses in stem cell research
• Global economic burden for society (BUSD)

(1) Aggregate of CNS diagnoses included in
graph. (2) Based on osteoarthrosis costs. (3)
Assumes that liver failure costs are maximum of
1/3 of all gastroenterology costs.
Note Year 2000 market figures Source Boston
Consulting Group, Swedish Brain Power, 2001
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Sweden possesses the required cross-functional
capabilities
(1) Includes e.g., hematologists, cardiologists,
transplant surgeons, orthopedics. Source Adapted
from Boston Consulting Group, Swedish Brain
Power, 2001
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Breakthroughs by Swedish researchers

• First clinical trials of fetal neural cell
  grafting in patients with Parkinsons disease
  (Anders Björklund and Olle Lindvall, 1987)
• Discovered the nesting gene, the most commonly
  used marker for neural stem cells (Urban Lendahl,
  1990)
• First to demonstrate that the human brain
  contains cells with stem cell-like properties
  (Peter Eriksson, 1998)
• First to identify adult neural stem cells capable
  of forming new neural stem cells (Jonas Frisén et
  al, 1999)
• First to identify adult stem cells potential to
  generate a variety of cells for other organs
  (Jonas Frisén et al, 2000)

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Commercialization resources in Sweden

• Basic research
• Funding
• Key competences
• Key technologies
• International and domestic networks with leading
  research groups
• Positive bioethical and regulatory environment

• Clinical development
• Good animal disease models for proof-of-concept
• High integration of basic and clinical research
• Multidisciplinary groups of relevant specialists
  with co-location/closeness
• Well-designed studies
• Excellent conditions for clinical research

• Commercialization
• Venture capital and management skills
• Technology transfer and spin-offs infrastructure
• Cluster of companies biotech, med-tech,
  diagnostics, big pharma
• Closeness including shared core facilities

Source Boston Consulting Group, Swedish Brain
Power, 2001
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Major sites for stem cell research

• Karolinska Institutet, Stockholm www.ki.se
• Leading position in basic research on adult stem
  cells, especially in neurology area.
• Very strong in developmental neurobiology and
  cellular biology.
• Good IVF clinic and leading position in embryonic
  stem cell lines development.
• Strong stem cell transplantation unit.
• Strong developmental biology groups in CNS,
  pancreas and hematology.
• Sahlgrenska Academy, Göteborg www.sahlgrenska.gu.
  se
• Leading position in establishing embryonic cell
  lines.
• Strong IVF program.
• Leading position in research on adult
  neurogenesis and stem cell neurobiology.
• Strong developmental biology groups in pancreas,
  blood vessel formation, and cellular biology.
• Leading position in developing FDA approved,
  (GMP) certified cell-based transplantation
  therapy (cartilage repair).
• Lund University, Lund www.lu.se
• Leading position in clinical applications of cell
  therapy in neurology area.
• Good stem cell biology in hematopoetic area and
  gene therapy vectors.

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Examples of stem cell research projects (1)

• Chalmers University of Technology, Göteborg
• Julie Gold Methods to steer differentiation of
  stem cells at specific spatial locations using
  cell surface interactions.
• Tomas Gustavsson Development of advanced image
  analysis systems to study cell division and
  differentiation.
• Sahlgrenska Academy, Göteborg
• Peter Eriksson Broad studies on the molecular
  mechanisms involved in neural stem cell
  proliferation and differentiation and their use
  in therapeutic strategies.
• Lars Hamberger/Charles Hanson Improving culture
  methods for blastocyst development to secure
  optimized stem cell material for further
  identification and analysis.
• Anders Lindahl Development, growth and
  regeneration of hyaline cartilage.
• Henrik Semb Definition of optimal cultivation
  conditions for human embryonic stem cells and
  continuous generation of such lines.
• University of Lund
• Anders Björklund Neural stem cells and
  immortalization of neural stem cell lines.
  Development of cells for transplantation.
• Sten Eirik Jacobsen Hematopoietic stem cells and
  molecular mechanisms governing stem cell
  differentiation.
• Stefan Karlsson Research program studying the
  genetic control of hematopoietic stem cell
  proliferation.
• Olle Lindvall Research program for
  transplantation of neural stem cells, for
  instance to regain function after Parkinsons
  disease and stroke.

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Examples of stem cell research projects (2)

• Karolinska Institutet, Stockholm
• Ernest Arenas Identification and
  characterization of signals required to instruct
  and promote the survival of midbrain dopaminergic
  (DA) neurons.
• Patrik Ernfors Neural crest stem cells and
  regulation of differentiation and functionality
  of peripheral sensory neurons.
• Jonas Frisén Broad neurobiological research
  studying the physiological importance of nerve
  cell development and regulating mechanisms.
• Outi Hovatta/Lars Ährlund-Richter Finding
  optimal cultivation conditions for human
  embryonic stem cells with particular regard for
  potential clinical applications.
• Katarina Le Blanc Human mesenchymal studies of
  stem cells from bone marrow of adult individuals
  and from fetal livers.
• Urban Lendahl Gene regulation in CNS stem cells,
  particularly in relation to the Notch signaling
  pathway.
• Thomas Perlman How signals from nuclear
  receptors affect the development and survival of
  certain neural stem cell types.
• Olle Ringdén Allogenic bone marrow or
  hematopoietic stem cell transplantation in the
  treatment of human blood disease, particularly
  leukemia.
• University of Linköping
• Per Fagerholm Effect of corneal stem cell
  transplantation to treat various forms of corneal
  damage. Strong clinical profile.

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Examples of stem cell research projects (3)

• University of Stockholm
• Barbara Cannon/Jan Nedergaard Study of brown fat
  tissue, with particular regard to the regulatory
  functions of noradrenaline.
• Anders Jakobsson Homologic recombination in
  embryonal mouse stem cells to produce mice with
  genetic mutations.
• University of Umeå
• Leif Carlsson Clarification of molecular
  mechanisms for immortalizing hematopoietic stem
  cells and development of systems for in-vitro
  cultivation of hematopoietic stem cells for
  clinical purposes.
• Swedish University of Agricultural Sciences,
  Uppsala
• Eva Hellmén Organogenesis of lymph nodes in
  relation to varying breast tumor phenotypes in
  differing species.
• University of Uppsala
• Michael Welsh The role of the endothelium in
  pancreatic beta cell differentiation.

Source Swedish Research Council, A Survey on
Stem Cell Research in Sweden, 2002
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Favorable Swedish legislative climateGuidelines
for ethical review of human stem cell research
applications

• Stem cells from adults YES
• Stem cells from umbilical-cord blood after
  childbirth YES
• Stem cells from aborted foetuses before week 14
  YES
• Stem cells from embryos which remain after in
  vitro fertilization YES
• Creation of embryos from eggs and sperm solely
  for research purposes NO
• Somatic cell nuclear transfer (therapeutic
  cloning) YES

While many countries are hampered by the ethical
debate to limit embryonic research, Sweden has
supported the development of potential benefits
instead. Elliott B. Davis, Next Wave Funds
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Making stem cell research a business

• Cell Therapeutics Scandinavia, Göteborg ?
  therapies for human diseases and other
  applications of stem cell technologies.
• Neuronova, Stockholm ? technologies to
  proliferate adult neural stem cells in culture
  and to differentiate them into dopaminergic
  neurons, for transplantation use w/ Parkinsons
  disease.
• NsGene, Copenhagen (Stockholm/Lund) ? products to
  treat neurological diseases, based on cell and
  gene therapy
• Vitrolife, Göteborg ? products for assisted
  reproduction and transplantation.

Target areas include neurodegenerative disorders,
degenerative joint diseases, cardiovascular
diseases, and diabetes.
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ISA your business facilitator

• Dedicated life sciences teams in the U.S, Europe
  and Asia.
• For further information, please contact Lars
  Vedin M.D. Head of Life Bio SciencesDirect
  phone [phone]Mobile phone [phone]
  73 90lars.vedin_at_isa.se www.isa.se/lifesciences.
  htm