About Us



Orphazyme was founded in 2009 with the objective to develop new therapies for patients suffering from protein-misfolding diseases with no or limited treatment options available.

The company was based on a scientific discovery on the function of Heat-Shock Proteins (HSPs) by Thomas Kirkegaard Jensen and Professor Marja Jäättelä that was published in the scientific journal Nature. Since inception, Orphazyme has translated the scientific discovery into a late-stage clinical development program.




To profoundly impact the lives of patients with orphan diseases and their families.


Bringing innovative therapies to patients with rare, serious diseases.




The strategy is to develop treatments for orphan diseases with protein misfolding where it can apply its specialized know-how in HSPs.   

The objective is to successfully conduct and complete the planned and on-going trials of arimoclomol for the treatment of protein-aggregation diseases, sporadic Inclusion Body Myositis (sIBM) and Amyotrophic Lateral Sclerosis (ALS), and the lysosomal storage diseases, Niemann Pick disease Type C (NPC) and Gaucher disease. 

As the clinical development program for arimoclomol progresses, Orphazyme intends to refine and finalize its commercialization strategy and build its commercial structure and operations. The company intends to build its own sales force in key markets. In markets outside the USA and Europe, Orphazyme currently intends to partner with local/regional distributors or license partners in certain other geographical areas. 

Protein misfolding is the hallmark of a broad range of diseases and Orphazyme’s strategy is to use its expertise, including proprietary know-how to select and develop new leads for suitable diseases, hence the development of a proprietary suite of New Molecular Entities (NMEs) with improved characteristics. Orphazyme intends to select diseases suitable for the NMEs based on genetic and mechanistic insights into selected protein-misfolding diseases. 



Core values

Care for people with rare diseases with a high unmet medical need.

Work tirelessly to conduct the most thorough, diligent research and bring results to the lives of patients as quickly and safely as possible.

Build powerful relationships with everyone connected to the diseases we are treating, to learn from the wider community.


Management Team

Kim Stratton.jpg
Kim Stratton
Chief Executive Officer

  • Joined in 2019
  • Board member of Novozymes A/S and Vifor Pharma
  • Previously at Shire Pharmaceuticals and Novartis
  • Thomas Blaettler.jpg
    Thomas Blaettler, MD
    Chief Medical Officer

  • MD, University of Zürich
  • Board certified neurologist
  • Joined in 2016
  • 12+ years' experience in neuroscience development
  • Previously at Roche, Bristol-Myers Squibb and Novartis
  • Thomas Kirkegaard Jensen.jpg
    Thomas Kirkegaard Jensen, PhD
    Chief Scientific Officer

  • PhD, in Medicine, University of Copenhagen
  • Co-founder
  • Orphazyme's scientific rationale published in Nature and ScienceTM with +200 references
  • Inventor of +60 patents in heat shock response therapy
  • PJM.jpg
    Paul Merrigan, MBA
    Chief Commercial Officer

  • MBA, Boston University
  • Joined in 2018
  • 20+ years’ experience in rare diseases in the biopharmaceutical industry
  • Previously at Catalyst Pharmaceuticals, Aegerion Pharmaceuticals, Genzyme, Genentech, Marion Laboratories and Pfizer
  • AFV.jpg
    Anders Vadsholt, MBA
    Chief Financial Officer

  • MBA in Finance, Melbourne University
  • Joined in 2016
  • 18+ years' biotech and corporate finance experience
  • Previously at Topotarget, Bankinvest Biomedical Venture, 7TM Pharma and Carnegie
  • Chief Executive Officer

    Anders Mørkeberg Hinsby, PhD

    "I co-founded Orphazyme with the aim to use my skills and experience to make a meaningful difference to the lives of those suffering from rare diseases. Having completed a PhD in medicine and worked for over 10 years in the life sciences investment industry, I lead Orphazyme with a focus on striving for excellence in every facet of the company."

    Chief Scientific Officer

    Thomas Kirkegaard Jensen, PhD

    "It’s my scientific research which forms the foundation of Orphazyme’s work. Having spent years wrestling cancer biology, one late night in 2007 I was looking down the microscope and realised that I had made a discovery that transcended cancer biology and could change the lives of patients suffering from a group of rare, devastating diseases. That's when I decided to found Orphazyme; to use my knowledge and skills to bring a novel therapy to patients suffering from rare diseases."

    Chief Financial Officer

    Anders Vadsholt

    “I bring successful orphan product approval experience and insight in late stage development. My area of expertise is private and public financing, prudent financial management and deal making. I’m excited about Orphazyme’s technology and anticipate our lead product will be commercially available within a few years.”

    Chief Medical Officer

    Thomas Blaettler, MD

    "As the CMO of Orphazyme I am thrilled to advance our programme in diseases that today have no or very limited treatment options and that inevitably lead to disability and in most cases to death. I build on my experience in clinical neurology and drug development to advance our ambitious clinical programme. My areas of expertise include neurology, psychiatry and rare diseases, and I’ve lead development programmes for new drugs from phase I to phase III."

    OUR Team



    Georges Gemayel.jpg
    Georges Gemayel
    Chairman of the board

  • Member since 2012
  • Chairman of nomination committee
  • Born 1960, American nationality
  • Georges Gemayel holds a Master's degree and a PhD degree in Pharmacology from Paris-Sud University and a Docteur d´Exercice en Pharmacie from the St. Joseph University
  • Bo Jesper Hansen.jpg
    Bo Jesper Hansen
    Deputy Chairman of the board

  • Member since 2010
  • Chairman of the remuneration committee
  • Born 1958, Danish nationality
  • Bo Jesper Hansen holds a MD and PhD degree in Medicine from the University of Copenhagen.
  • Martin Bonde.jpg
    Martin Bonde

  • Member since 2010
  • Member of nomination committee
  • Born 1963, Danish nationality
  • Martin Bonde holds a Graduate Diploma in Business Administration (HD i Udenrigshandel) from Copenhagen Business School, a Master of Science and a PhD in Chemical Engineering from the Technical University of Denmark.
  • Remi Droller.jpg
    Rémi Droller

  • Member since 2015
  • Member of the remuneration committee
  • Born 1975, French nationality
  • Rémi Droller holds a master’s degree in molecular biology from Université Pierre et Marie Curie and a master’s degree in finance and management of innovation from Masternova.
  • Sten Verland.jpg
    Sten Verland

  • Member since 2010
  • Member of the nomination committee
  • Member of the audit committee
  • Born 1957, Danish nationality
  • Sten Verland holds a master’s degree in Biology and Mathematics and a PhD in Immunology, both from the University of Copenhagen.
  • Martijn Kleigwegt.jpg
    Martijn Kleijwegt

  • Member since 2017
  • Member of the audit committee
  • Born 1955, Dutch nationality
  • Martijn Kleijwegt holds a master’s degree from the University of Amsterdam.
  • Anders Hedegaard.jpg
    Anders Hedegaard

  • Member since 2017
  • Member of the remuneration committee
  • Born 1960, Danish nationality
  • Anders Hedegaard holds a Master of Science in Chemical Engineering and Biochemistry from the Technical University of Denmark.
  • Catherine Moukheibir.jpg
    Catherine Moukheibir

  • Member since 2017
  • Chairman of the audit committee
  • Born 1959, American, Lebanese and British nationality
  • Catherine Moukheibir holds a Master in Economics and an MBA degree, both from Yale University.
  • Chair (Independent)

    Georges Gemayel

    • Currently, Chairman of the Board of Enterome and Oxthera
    • Director at Dimension Therapeutics, Momenta, Supernus and Raptor Pharmaceuticals
    • Previously, General Manager at Roche Portugal; Vice Presidentat Specialty Care Roche US; Executive Vice President at Genzyme; CEO of Altus; Director of Adolor, NPS, Epitherapeutics and Prosensa; Executive Chairman at FoldRx and Syndexa


    Bo Jesper Hansen

    • Executive Chairman of the board at public company Swedish Orphan Biovitrum
    • Founder of Scandinavian Medical Research through which he contracted as a medical advisor for Pfizer, Yamanouchi and Synthelabo
    • Various executive positions in Swedish Orphan International AB since 1993 (CEO in the period 1998-2010)
    • Board member of Ablynx, Newron, CMC and Karolinska Development AB (Chairman)

    Martijn Kleijwegt

    • Currently a Managing Partner and founder of LSP. His prime focus and responsibility within LSP is to invest in unlisted securities and the general management of the firm
    • Board member of Kiadis Pharma, Orphazyme, Oxthera and Pharvaris
    • Prior to establishing LSP, Martijn ran another investment firm where he was responsible for investments in the healthcare sector
    • Among others, Martijn served as a Director of Prosensa (NL, IPO 2013, sold to Biomarin), Movetis (BEL, IPO in 2009, sold to Shire), Crucell (NL, IPO in 2000, sold to Johnson & Johnson), Rhein Biotech (NL, IPO in 1999, sold to Berna Biotech), and Qiagen (GER, IPO in 1996)


    Martin Bonde

    • CEO Vaccibody AS
    • Serial entrepreneur. Founded and managed several companies in Denmark and USA (EpiTherapeutics ApS, NatImmune A/S, Arpida A/S, Combio A/S, CelTor Inc., Torsana Biosensor)
    • Fronted several successful exits over the last decade (e.g. EpiTherapeutics ApS to Gilead, NatImmune A/S to IPC; Combio A/S to Arpida AG; and Torsana Biosensor A/S to Celtor Inc)
    • Experience heading out-licensing, acquisitions, in-licensing etc.

    Martin Kornum

    • Co-founder of Orphazyme
    • Currently: European Patent Attorney at Coloplast A/S, Denmark
    • Previously, Patent Agent at Davis Wright Tremaine LLP, San Francisco

    Nanna Luneborg

    • Principal, Novo Ventures
    • Member of the board of directors of E3Bio, Glionova, Inthera, ObsEva and Orphazyme
    • Spent four years with Novo Seeds, building a strong portfolio of seed and Series A stage companies, before joining Novo Ventures in 2016
    • Previously: Apposite Capital
    • PhD in Neuroscience from University College London, an MBA with distinction from University of Cambridge, and a 1st class BA from University of Oxford

    Patrick Krol

    • Managing Partner of Aescap Venture
    • Serves on the BoDs of Orphazyme ApS and Shire Pharmaceuticals International Licensing
    • Serves on the supervisory boards of i-Optics, to-BBB, and f-star
    • Founder and Managing Director of Firm United Healthcare and co-founder of the two healthcare ventures Interactive Healthcare and the Healthcare Management School; Previously, Director of Healthcare of Fleishman-Hillard, UK
    • Visiting teacher at the business school of the University of Wageningen, NL

    Remi Droller

    • Managing Partner of Kurma Partners
    • Currently: MS in molecular biology (Paris VI University) and MS in finance and Innovation Management (Masternova – AgroPariTech). Rémi joined Kurma in September 2010. He was previously with CDC Innovation from 2000 to 2003 and with AGF Private Equity (now Idinvest Partners) from 2003 to 2010 where he was in charge of the development of the Life Sciences investment activities. During this period, Rémi served on the board of several investee companies such as Adocia, Bioalliance Pharma (listed on Euronext), Domain Therapeutics, Integragen (listed on Alternext), Novagali Pharma (listed on Euronext), Prosensa and Zeland Pharma.

    Sten Verland

    • Partner and co-founder at Sunstone Capital A/S
    • Serves on the BoD of Orphazyme ApS, Zymenex A/S (chairman), Action Pharma A/S (chairman), F2G Ltd., Scanbur A/S, StemCare A/S, and Vaximm AG
    • Serial biotech entrepreneur. Has been involved in the start-up and management of several companies including Synarc Inc., M&B A/S (now Taconic Europe), and StemCare A/S
    • Previously: VP General Manager Europe at Synarc, Inc.; CEO and co-owner at M&B A/S
    • Postdoctoral fellow and Ph.D. in Immunology at the University of Copenhagen

    Novo Ventures

    Novo A/S, the holding company in the Novo Group, was established prior to the demerger of Novo Nordisk A/S in 2000. Novo A/S is a private limited liability company fully owned by the Novo Nordisk Foundation. Besides being the majority shareholder in Novo Nordisk A/S and Novozymes A/S, Novo A/S provides seed capital (through Novo Seeds), venture capital (through Novo Ventures), and growth capital (through Novo Growth Equity) to development stage companies within life science and biotechnology. Novo A/S also manages a broad portfolio of financial assets.

    Sunstone Capital

    Sunstone Capital is an independent venture capital investor founded in 2007 by an international team of industry experts with combined entrepreneurial, operational and financial experience. Sunstone Capital focuses on developing and expanding early-stage Life Science and Technology companies with strong potential to achieve global success in their markets. Sunstone Capital currently manages four Life Science funds and three Technology funds. Within Life Science, Sunstone Life Science Ventures has invested in more than 35 companies in the areas of pharmaceuticals, medical technologies and diagnostics, and has completed several successful exits and IPOs. Managing total funds of EUR 693 million, Sunstone Capital is one of the largest and most active European venture capital investors.

    Aescap Venture

    Aescap Venture is a venture capital company investing in private medical companies in Europe. Aescap Venture has a long-term investment horizon and invests in high-potential companies. This involves investments in all phases of development with an emphasis on earlier stages. Aescap Venture's added value is based on a multi-disciplinary team of experienced investment partners with proven track records of success and the skills to coach entrepreneurs in accelerating the growth of their companies.

    Kurma Partners

    Kurma Partners is a key European player in the financing of Innovation in Healthcare and Biotechnology, from pre-seed to growth capital, notably through Kurma Biofund I and II, and strategic partnerships with prestigious European research and medical institutions. Kurma Biofund II is the first Venture Capital fund dedicated to financing innovation in the rare diseases space.


    LSP is an independent European investment firm, providing financing for private and public life sciences companies. LSP’s mission is to connect investors to inventors, focusing on unmet medical needs. Since the late 1980s, LSP’s management has invested in about 100 innovative enterprises, many of which have grown to become leaders of the global life sciences industry. With over €1 billion of investment capital raised to date and offices in Amsterdam, Munich and Boston, LSP is one of Europe’s leading life sciences investors. The LSP Health Economics Fund invests in innovative products that can increase the quality of health care, while reducing the cost of care.


    The ALS Investment Fund supports and finances biotech companies that develop drugs and diagnostics for Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease. The Fund was initiated by 3 Dutch ALS patients and is supported by a top-tier European VC firm, Life Sciences Partners. The ALS Investment Fund invests globally, but focuses on Europe and the US. The fund brings depth of disease-specific knowledge and a deep network in the space.