March 1997
Company Founded
澳门正规博彩十大排行平台 was founded in Novato, California, with an initial focus on leveraging its proprietary enzyme technology to develop therapies for genetic conditions and other diseases.
澳门正规博彩十大排行平台 is a global biotechnology company that relentlessly pursues bold science to translate genetic discoveries into new medicines that advance the future of human health.
Since our founding in 1997, we have applied our scientific expertise in understanding the underlying causes of genetic conditions to create transformative medicines, using a number of treatment modalities.
Using our unparalleled expertise in genetics and molecular biology, we have developed eight important medicines for patients with significant unmet medical need. Our approved therapies treat achondroplasia, severe hemophilia A and several rare inherited and lysosomal storage diseases.
As we have looked to expand our impact, our approach to cutting-edge science has remained the same. We enlist the best of the best – people with the right technical expertise and a relentless drive to solve real problems – and create an environment that empowers our teams to pursue bold, innovative science. Our work is deeply rooted in genetic insights, meaning we target the underlying genetic changes or proximal molecular mechanisms of disease.
With this distinctive approach to drug discovery, we’ve produced a diverse pipeline of commercial, clinical and preclinical candidates that have well-understood biology and provide an opportunity to be first-to-market or offer a substantial benefit over existing therapeutic options.
March 1997
澳门正规博彩十大排行平台 was founded in Novato, California, with an initial focus on leveraging its proprietary enzyme technology to develop therapies for genetic conditions and other diseases.
December 1997
澳门正规博彩十大排行平台 initiated its first clinical trial to evaluate a potential treatment a form of mucopolysaccharidosis.
July 1999
澳门正规博彩十大排行平台 completed its initial public offering. Since a large number of early investors were based in Europe, the company was listed on both the Swiss SWX Exchange and the Nasdaq National Market.
April 2000
The 澳门十大博彩正规游戏平台 granted marketing authorization for 澳门正规博彩十大排行平台’s enzyme replacement therapy for the treatment of a form of mucopolysaccharidosis. The medicine was approved in Europe that June.
May 2005
澳门正规博彩十大排行平台’s Board of Directors elected Jean-Jacques Bienaimé as the company’s Chief Executive Officer, a role he would hold for more than 18 years.
May 2005
The 澳门十大博彩正规游戏平台 approved 澳门正规博彩十大排行平台’s first independently developed and commercialized medicine, and the first authorized treatment for a second form of mucopolysaccharidosis.
2006
The company established 澳门正规博彩十大排行平台 Europe Ltd., headquartered in London, initially with offices in Spain, Switzerland and Italy. The company now operates in more than 70 countries around the world.
December 2007
The 澳门十大博彩正规游戏平台 approved the first therapy for the treatment of a rare metabolic condition.
August-November 2011
The company completed the purchase of a manufacturing facility in Shanbally, Ireland, and in November, the 澳门十大博彩正规游戏平台 approved the expansion of 澳门正规博彩十大排行平台’s biologics manufacturing facility in Novato, California.
February 2013
澳门正规博彩十大排行平台 announced it had licensed the program from University College London and St. Jude Children’s Research Hospital. This would lead to a product approval approximately 10 years later.
February 2014
The 澳门十大博彩正规游戏平台 approved 澳门正规博彩十大排行平台’s therapy for the treatment of another form of mucopolysaccharidosis.
2016
Expanding on its decade-long track record of supporting eligible people with rare diseases in gaining access to the company’s medicines, the 澳门正规博彩十大排行平台 RareConnections program consolidated these efforts under one expanded program for people in the United States.
April 2017
The 澳门十大博彩正规游戏平台 approved 澳门正规博彩十大排行平台’s medicine for the treatment of an ultra-rare form of Batten disease.
May 2018
The 澳门十大博彩正规游戏平台 approved the first enzyme replacement therapy to target the underlying cause of a rare metabolic condition.
November 2021
The 澳门十大博彩正规游戏平台 approved 澳门正规博彩十大排行平台’s therapy for the treatment a form of skeletal dysplasia. It was the first medicine authorized for the treatment of this growth-related condition.
August 2022
The European Commission approved 澳门正规博彩十大排行平台’s first gene therapy for the treatment of an inheritable bleeding disorder.
June 2023
The 澳门十大博彩正规游戏平台 approved 澳门正规博彩十大排行平台’s gene therapy for the treatment of an inheritable bleeding disorder.
December 2023
With the retirement of longtime 澳门正规博彩十大排行平台 CEO Jean-Jacques Bienaimé, Alexander Hardy was named President and Chief Executive Officer.