We celebrate the people we serve
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 are better together
In the face of obstacles, we see opportunity
We aim higher
News and events
In an effort to bring us closer together, we want to keep you informed and bring you the latest news from the BioMarin Hemophilia team.
6/21/19-6/23/19 Grapevine, TX | Lone Star Chapter of the NHF and Texas Central Hemophilia Foundation Texas Bleeding Disorders Conference |
6/22/19 Grapevine, TX | Breaking Through 2019 Art Therapy Workshop at the Texas Bleeding Disorder Conference |
7/6/19-7/10/19 Melbourne, Australia | ISTH 2019 Congress: BioMarin is presenting 3 platform presentations, a symposium and a poster |
7/26/19-7/28/19 Fort Lauderdale, FL | Breaking Through 2019 Art Therapy Workshop with the Florida Hemophilia Association |
9/14/19 Portland, OR | Breaking Through! Workshop with the Hemophilia Foundation of Oregon
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10/3/19-10/5/19 Anaheim, CA | NHF Conference |
10/26/19-10/27/19 Warwick, RI | Breaking Through 2019 Art Therapy Workshop with the New England Hemophilia Association |
12/7/19 Las Vegas, NV | Breaking Through 2019 Art Therapy Workshop with the Nevada Chapter of NHF |
01/20/20 Chicago, IL | Breaking Through 2019 Art Therapy Workshop with Bleeding Disorders Alliance of Illinois |
BioMarin and hemophilia timeline
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Click along the timeline and follow the journey
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Hemophilia—
The royal disease1800s
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.
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Mendel’s theory
of inheritance1865
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.
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DNA isolated
1869
Frederick Miescher isolated DNA from cells for the first time and called it nuclein.
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Whole-blood transfusion
in hospital1940s
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.
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DNA double helix
described1953
James D. Watson and Francis H. Crick described the double helix structure of DNA. They received the Nobel Prize for their work in 1962.
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At-home replacement therapy
available, with plasma-derived
factor concentrates1970s
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.
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Dr. Barrie Carter begins
work on AAV-mediated gene transfer biotechnology at the National Institutes of Health1970
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Dr. Gordon Vehar begins
work on factor VIII1976
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Genes for factor VIII
and factor IX are cloned1980s
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.
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The Centers for Disease
Control (CDC) reports first AIDS
case in hemophilia1982
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.
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Dr. Barrie Carter publishes
a paper describing the use
of AAV as a vector1984
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.
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Dr. Gordon Vehar publishes
a paper reporting successful
factor VIII cloning1984
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.
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Dr. Wing Yen Wong
begins hematology
fellowship1987
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First gene therapy
trial in humans1990
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.
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Recombinant clotting
factors approvedFVIII 1992
FIX 1997
FVIIa 1999Throughout 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.
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BioMarin incorporates
1997
Founded in March 1997, with a $1.5 million investment from Glyko Biomedical Ltd., BioMarin was open for business.
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Lessons learned regarding risks
related to potential for severe
immune response in early gene
therapy trial with non-AAV vector1999
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.
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Prophylaxis is recommended as
standard of care2000s
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Human Genome Project
completed2003
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.
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First gene therapy trial in
hemophilia B using
AAV vector technology2005
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.
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BioMarin therapies for
rare diseases approvedMucopolysaccharidosis I 2003
Mucopolysaccharidosis VI 2005
Phenylketonuria 2007Over the years, BioMarin has extended its commitment to those communities with rare diseases.
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Dr. Gordon Vehar joins BioMarin
2008
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Dr. Barrie Carter joins BioMarin
2011
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Extended half-life
factors approved2014
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BioMarin therapies for
rare diseases approvedMucopolysaccharidosis IVA 2014
Neuronal ceroid lipofuscinosis type 2 2017 -
Investigational gene therapy by BioMarin: Hemophilia A trial to research safety and efficacy starts (BMN 270-201)
2015
After years of scientific study and collaboration with world experts, in 2015, BioMarin began the BMN 270-201 clinical trial—evaluating the safety, proper dosing, and efficacy of a gene therapy candidate for adults with severe hemophilia A.
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Dr. Wing Yen Wong joins BioMarin
2016
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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 timely 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.