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The Sound of What's Next in Hemophilia

You spoke, we listened. Hear the voices that are speaking out
for change in the hemophilia community.

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Voices of the Community

At BioMarin Hemophilia, we believe the sound of what’s next in hemophilia is you. Listen to some of the personal stories from parents, mentors, students, and leaders in the hemophilia community who inspire us to continue to look forward.

Get to know BioMarin Hemophilia

We’re here to amplify your voice

At BioMarin Hemophilia we aspire to lift the voice of the hemophilia community the same way you inspire us to keep researching and moving toward the exceptional with our science. Every day we make it our purpose to put you first. From our clinical research and development to supporting your local advocacy group—we’re focused on doing everything we can to hear and help the hemophilia community thrive.

What you can expect from us

Our values and purpose—they're not just words. Sure, they're on posters in our offices, right next to pictures of our kids and our dogs, but that's because we are driven by them. And we know we must live them, day in, day out, to effect real change.

Our purpose

We are inspired by a hemophilia community with huge aspirations and driven to deliver on the ingenuity of gene therapy clinical research and exploration.

Our behaviors

We celebrate the people we serve
We are better together
In the face of obstacles, we see opportunity
We aim higher

BioMarin and hemophilia timeline

  1. Hemophilia—
    The royal disease

    1800s

    Hemophilia is sometimes referred to as “the royal disease,” because it affected the royal families of England, Germany, Russia, and Spain in the 19th and 20th centuries.

    More

  2. Mendel’s theory
    of inheritance

    1865

    Born in the Old Austrian empire, Gregor Mendel was a Czech monk who worked out the basic laws of inheritance before the term gene had been invented. Mendel performed thousands of experiments with garden peas. He explained his results by describing two laws of inheritance that introduced the idea of dominant and recessive traits.

    More

  3. DNA isolated

    1869

    Frederick Miescher isolated DNA from cells for the first time and called it nuclein.

  4. Whole-blood transfusion
    in hospital

    1940s

    In 1940, Edwin Cohn, a professor at Harvard Medical School, developed the process of breaking down plasma into components and products. Albumin, a protein with powerful osmotic properties, plus gamma globulin and fibrinogen were isolated and became available for clinical use.

    In 1941, Isodor Ravdin, a Philadelphia surgeon, effectively treated victims of the Pearl Harbor attack with Cohn's albumin for shock.

    More

  5. DNA double helix
    described

    1953

    James D. Watson and Francis H. Crick described the double helix structure of DNA. They received the Nobel Prize for their work in 1962.

  6. At-home replacement therapy
    available, with plasma-derived
    factor concentrates

    1970s

    The modern management of hemophilia really began in the 1970s with the production of coagulation factors. This innovation greatly improved the quality of life and longevity of people with hemophilia, permitting the widespread adoption of at-home replacement therapy.

    More

  7. Dr. Barrie Carter begins
    work on AAV-mediated gene transfer biotechnology at the National Institutes of Health

    1970

  8. Dr. Gordon Vehar begins
    work on factor VIII

    1976

  9. Genes for factor VIII
    and factor IX are cloned

    1980s

    The successful cloning of the factor VIII gene in 1984 was a major breakthrough, allowing production of recombinant human factor VIII. Cloning of factor IX was first reported in 1982.

    More

  10. The Centers for Disease
    Control (CDC) reports first AIDS
    case in hemophilia

    1982

    In 1982, products derived from contaminated blood led to the first CDC-reported cases of patients with hemophilia A developing pneumonia and other infections that met the case definition of AIDS.

    More

  11. Dr. Barrie Carter publishes
    a paper describing the use
    of AAV as a vector

    1984

    In October 1984, Dr. Barrie Carter published A Human Parvovirus, Adeno-Associated Virus, as a Eucaryotic Vector: Transient Expression and Encapsidation of the Procaryotic Gene for Chloramphenicol Acetyltransferase, an article first describing the use of adeno-associated virus as a vector.

    More

  12. Dr. Gordon Vehar publishes
    a paper reporting successful
    factor VIII cloning

    1984

    Expression of Active Human Factor VIII From Recombinant DNA Clones by Dr. Gordon Vehar and his colleagues reported on the successful cloning of factor VIII, a breakthrough advancement.

    More

  13. Dr. Wing Yen Wong
    begins hematology
    fellowship

    1987

  14. First gene therapy
    trial in humans

    1990

    A 4-year-old girl suffering from adenosine deaminase (ADA), a condition in which people cannot resist common infections, became the first person to receive a non-AAV gene therapy treatment.

    More

  15. Recombinant clotting
    factors approved

    FVIII 1992
    FIX 1997
    FVIIa 1999

    Throughout the 1990s, synthetic factor products were manufactured using recombinant technologies. In 1992, the first recombinant factor VIII product was approved by the FDA. In 1997, the first recombinant factor IX product was granted FDA approval. In 1996, rFVIIa was approved in the European Union and in 1999 in the United States for use as a bypassing agent in patients with hemophilia A or B (factor VIII or factor IX deficiency) and an antibody inhibitor.

    More

  16. BioMarin incorporates

    1997

    Founded in March 1997, with a $1.5 million investment from Glyko Biomedical Ltd., BioMarin was open for business.

  17. Lessons learned regarding risks
    related to potential for severe
    immune response in early gene
    therapy trial with non-AAV vector

    1999

    Lessons were learned from serious problems that highlighted the potential toxicity of other viral vectors in earlier trials of gene therapy, leading to the research into new and different technologies.

    More

  18. Prophylaxis is recommended as
    standard of care

    2000s

  19. Human Genome Project
    completed

    2003

    The Human Genome Project (HGP) was an international research effort to sequence and map all of the genes—together known as the genome—of members of our species, homo sapiens. Completed in April 2003, the HGP gave us the ability, for the first time, to read nature's complete genetic blueprint for building a human being.

    More

  20. First gene therapy trial in
    hemophilia B using
    AAV vector technology

    2005

    In a 2005 clinical gene transfer trial, it was found that the human liver can be transduced by recombinant adeno-associated viral (rAAV) vector after in vivo delivery of the vector.

    More

  21. BioMarin therapies for
    rare diseases approved

    Mucopolysaccharidosis I 2003
    Mucopolysaccharidosis VI 2005
    Phenylketonuria 2007

    Over the years, BioMarin has extended its commitment to those communities with rare diseases.

  22. Dr. Gordon Vehar joins BioMarin

    2008

  23. Dr. Barrie Carter joins BioMarin

    2011

  24. Extended half-life
    factors approved

    2014

  25. BioMarin therapies for
    rare diseases approved

    Mucopolysaccharidosis IVA 2014
    Neuronal ceroid lipofuscinosis type 2 2017

  26. BioMarin doses the first patient in an investigational gene therapy trial in hemophilia A to research safety and efficacy
    (BMN 270-201)

    2015

    In 2015 BioMarin dosed its first patient in the BMN 270 clinical trial - evaluating the safety, dosing, and efficacy of a gene therapy for adults with hemophilia A.

  27. Dr. Wing Yen Wong joins BioMarin

    2016

Full Timeline
  1. 1800
  2. 65
  3. 69
  4. 1940
  5. 1953
  6. 1970
  7. 70
  8. 76
  9. 1980
  10. 82
  11. 84
  12. 84
  13. 87
  14. 1990
  15. 92
  16. 97
  17. 99
  18. 2000
  19. 03
  20. 05
  21. 07
  22. 08
  23. 2011
  24. 14
  25. 14
  26. 15
  27. 16

Connect and be heard

We believe in deep-rooted connections, two-way conversations, and open dialogue. So, we promise no robo-calls, no dinnertime chats, and no spam emails with smiling happy people wearing backpacks. You'll get information about what we’re hearing from the community, where we’ll be so we can connect, and how we’re amplifying your voice in great ways. CONNECT WITH US
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Connect and be heard

We believe in deep-rooted connections, two-way conversations, and open dialogue. So, we promise no robo-calls, no dinnertime chats, and no spam emails with smiling happy people wearing backpacks. You’ll get information about what we’re hearing from the community, where we’ll be so we can connect, and how we’re amplifying your voice in great ways.

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Due to compliance with other countries’/regions’ data-privacy laws, eg, the EU’s General Data Protection Regulation (GDPR), we are not collecting personal information from individuals outside of the US.

If you live in the EU, please visit BioMarinHemophilia.eu for updates.





THANK YOU

We believe that what's next in hemophilia starts with your voice—and we're so thankful to get to connect with you.

We promise to stay in touch and keep you updated on all things BioMarin Hemophilia.

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BioMarin Hemophilia