BioMarin Archives

In Depth 10 Jul 2024

Biotech’s battle against skeletal dysplasia: Ray of hope for children with rare genetic disorder

As treatments for skeletal dysplasia are intended to alleviate painful symptoms, two drugs are battling out in the clinic to prove their effectiveness.

July 10, 2024 - 7 minutesmins - By Roohi Mariam Peter

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 (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The […]

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 (valoctocogene roxaparvovec) treats the congenital factor 8 deficiency in adults patients without a history of factor VIII inhibitors without detectable antibodies to adeno-associated virus serotype […]

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, regulatory, and commercial milestone payments and a percentage of royalties on later sales. No financial details of the collaboration were revealed by either company. “We […]

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 Rare Diseases (EURORDIS) is once again coordinating Rare Disease Day, which takes place on the last day of February every year. The aim is to raise awareness of […]

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 is about. Organized by the banking firm of the same name, it is the 34th edition this year. And with age, comes exclusivity: The J.P. […]

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 in the States; Flagship Ventures. Flagship Ventures has yet again shown its comprehensive portfolio in biotech through another EU inspired Biotech start-up. Rubius Therapeutics in […]

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 trial for children with Sanfilippo Type B (MPS-III) in collaboration with France’s Institut Pasteur. UniQure is the Amsterdam gene-therapy biotech with a lot going on. Last year […]

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 biosimilar joint venture: WSJ Samsung is also interested in Biotech and more precisely in Biosimilars. It created a joint-venture with Biogen and now wants to […]

August 27, 2015 - 2 minutesmins - By Philip Hemme

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