BioMarin Archives

News and Trends 30 Jun 2023

FDA approves BioMarin gene therapy for hemophilia A

BioMarin Pharmaceutical Inc. has announced that the United States Food and Drug Administration (FDA) has approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A…

June 30, 2023 - 5 minutesmins - By Jim Cornall

News and Trends 26 Aug 2022

First gene therapy for adults with severe hemophilia approved by EC

The European Commission (EC) has granted conditional marketing authorization for a gene therapy to treat severe hemophilia A in adults it was announced yesterday (August 25). BioMarin Pharmaceutical Inc.’s Roctavian…

August 26, 2022 - 3 minutesmins - By Liza Laws

gene therapy cardiac rare disease biomarin dinaqor

News and Trends 7 May 2020

BioMarin Partners Swiss Biotech to Develop Cardiac Gene Therapies

US biotech BioMarin has partnered Swiss startup DiNAQOR to develop gene therapies to treat rare, inherited cardiac diseases. DiNAQOR will initially receive an upfront payment from BioMarin, followed by developmental,…

May 7, 2020 - 2 minutesmins - By Helen Albert

News and Trends 28 Feb 2017

It’s Global Rare Disease Day! What Cures is Biotech Cooking Up?

We've compiled a list of some of the latest advances of biotech in the field of rare disease to contribute to Rare Disease Day 2017. The European Organization for…

February 28, 2017 - 5 minutesmins - By Clara Rodríguez Fernández

jp morgan biotech san francisco california 2016

News and Trends 14 Jan 2016

The Biotech World is Buzzing: J.P. Morgan’s 2016 Phenomenon Kicks off this Week!

Imagine a conference where all the Biotech CEOs and investors of the World come together...all the major players in this Industry, under one roof. That's basically what the J.P.Morgan Conference…

January 14, 2016 - 4 minutesmins - By Philip Hemme

blood cells transport drugs rubius therapeutics flagship erytech pku

News and Trends 14 Dec 2015

Red Blood Cells to Carry Drugs? A US Start-Up Inspired by French Biotech to Focus on Rare Metabolic Disease

Erytech (France) is working on a drug-transportation system using red blood cells. Now US Rubius Therapeutics joins Erytech on this new approach to drug delivery, backed by the huge VC…

December 14, 2015 - 3 minutesmins - By Dani Bancroft

News and Trends 22 Sep 2015

UniQure’s cure for rare Lysosomal disease ready for phase III trial

UniQure from the Netherlands has promising results for its enzyme replacement therapy phase Ib/II trial targeting a rare Lysosomal disease. This has granted fast-track approval to launch its phase III…

September 22, 2015 - 3 minutesmins - By Dani Bancroft

News and Trends 27 Aug 2015

The 5 things outside Europe you need to know this week (Samsung $1Bn Biotech IPO, Microbiome blindness, Google Biotech)

Hi there! Here are the 5 most important Biotech things that happened outside Europe in the last few days. Enjoy and share! Samsung, Biogen lining up a $1B IPO for…

August 27, 2015 - 2 minutesmins - By Philip Hemme

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