BioMarin Pharmaceutical

July 13, 2018July 13, 2018

Companies Collaborating to Conduct Musical Workshop for Teens Affected by Bleeding Disorders

News

BioMarin Pharmaceutical Inc. is partnering with digital content agency Believe Limited to produce a three-day musical workshop for young members of the bleeding disorders community. The “Breaking Through!” musical theater intensive, ... Read more

May 16, 2018May 16, 2018

Valoctocogene Roxaparvovec Study Doses 1st Hemophilia Patient with Pre-existing AAV5 Antibodies

News

A Phase 1/2 trial (NCT03520712) testing BioMarin Pharmaceutical’s investigational gene therapy valoctocogene roxaparvovec in severe hemophilia A patients with pre-existing AAV5 antibodies has dosed its first participant. “Administration of valoctocogene ... Read more

November 1, 2017November 1, 2017

BioMarin’s Investigative Gene Therapy for Hemophilia A Wins FDA Breakthrough Designation

News

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to BioMarin Pharmaceutical’s investigative therapy, valoctocogene roxaparvovec to treat patients with hemophilia A. This new status is likely to ... Read more

October 27, 2017

FDA Grants Investigational New Drug Status for BioMarin’s BMN 270 Hemophilia A Therapy

News

The U.S. Food and Drug Administration (FDA) has approved BioMarin Pharmaceutical‘s Investigational New Drug (IND) application for its gene therapy candidate BMN 270 to treat severe hemophilia A. FDA’s decision ... Read more

August 9, 2017

Biomarin Plans to Test Two Doses of BMN 270 in Phase 3 Trials for Treatment of Hemophilia A

News

BioMarin Pharmaceutical will add an additional Phase 3 study to the clinical development of its investigational gene therapy BMN 270 for hemophilia A. BioMarin will conduct two separate trials testing ... Read more

June 22, 2016

BioMarin to Present Phase 1/2 Data for BMN 270 at 2016 Hemophelia Congress

News

San Rafael, California-based BioMarin Pharmaceutical will present interim data of an open-label, Phase 1/2 clinical trial of BMN 270, an investigational gene therapy treatment for hemophilia at the upcoming XXXII International ... Read more

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