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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.
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.
1869
Frederick Miescher isolated DNA from cells for the first time and called it nuclein.
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.
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.
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.
1970
1976
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.
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.
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.
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.
1987
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.
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.
1997
Founded in March 1997, with a $1.5 million investment from Glyko Biomedical Ltd., BioMarin was open for business.
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.
2000s
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.
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.
Mucopolysaccharidosis I 2003
Mucopolysaccharidosis VI 2005
Phenylketonuria 2007
Over the years, BioMarin has extended its commitment to those communities with rare diseases.
2008
2011
2014
Mucopolysaccharidosis IVA 2014
Neuronal ceroid lipofuscinosis type 2 2017
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.
2016
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