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.
We are inspired by a hemophilia community with huge aspirations and driven to deliver on the ingenuity of gene therapy clinical research and exploration.
We are better together
In the face of obstacles, we see opportunity
We aim higher
Speak Out Summits
What is a SPEAK OUT SUMMIT?
We believe real change happens when we bring the right people together. Speak Out Summits are our way of connecting, listening, and collaborating with the hemophilia community. Think of it as a choir of voices from all around the bleeding disorders community coming together—digging into some really tough challenges and then figuring out how to solve them.
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.
|11/3/18-11/4/18 Manchester, NH||
NEHA Fallfest Education Event with the New England Hemophilia Association
|11/11/18 Oakland, CA||
HFNC Family Education Day with the Hemophilia Foundation of Northern California
|11/12/18 New York, NY||
Breaking Through with Believe Limited and Patrick Lynch
|11/18/18 New York, NY||
Family Education Day with the New York City Hemophilia Chapter
BioMarin and hemophilia timeline
Click along the timeline and follow the journey
The royal disease
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
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
Frederick Miescher isolated DNA from cells for the first time and called it nuclein.
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
DNA double helix
James D. Watson and Francis H. Crick described the double helix structure of DNA. They received the Nobel Prize for their work in 1962.
At-home replacement therapy
available, with plasma-derived
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
Dr. Barrie Carter begins
work on AAV-mediated gene transfer biotechnology at the National Institutes of Health
Dr. Gordon Vehar begins
work on factor VIII
Genes for factor VIII
and factor IX are cloned
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
The Centers for Disease
Control (CDC) reports first AIDS
case in hemophilia
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
Dr. Barrie Carter publishes
a paper describing the use
of AAV as a vector
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
Dr. Gordon Vehar publishes
a paper reporting successful
factor VIII cloning
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
Dr. Wing Yen Wong
First gene therapy
trial in humans
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
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
Founded in March 1997, with a $1.5 million investment from Glyko Biomedical Ltd., BioMarin was open for business.
Lessons learned regarding risks
related to potential for severe
immune response in early gene
therapy trial with non-AAV vector
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
Prophylaxis is recommended as
standard of care
Human Genome Project
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
First gene therapy trial in
hemophilia B using
AAV vector technology
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
BioMarin therapies for
rare diseases approved
Mucopolysaccharidosis I 2003
Mucopolysaccharidosis VI 2005
Over the years, BioMarin has extended its commitment to those communities with rare diseases.
Dr. Gordon Vehar joins BioMarin
Dr. Barrie Carter joins BioMarin
BioMarin therapies for
rare diseases approved
Mucopolysaccharidosis 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)
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.More
Dr. Wing Yen Wong joins BioMarin
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.