18:13 , Dec 8, 2017 |  BioCentury  |  Product Development

Making of Mepsevii

It may take a few years for Ultragenyx Pharmaceutical Inc. to recoup its investment in Mepsevii vestronidase alfa-vjbk, but the company expects to continue to reliably churn out treatments for ultra-rare diseases alongside a portfolio...
07:00 , Mar 28, 2016 |  BioCentury  |  Strategy

Canbridge can-can

Canbridge Life Sciences Ltd .'s deal with Aveo Pharmaceuticals Inc. will see the Chinese biotech developing its first programs outside Asia. But Chairman and CEO James Xue says the company is not deviating from its...
08:00 , Dec 8, 2014 |  BioCentury  |  Strategy

BioMarin bulks up

Five years after discussing a licensing deal with Prosensa Holding N.V. , BioMarin Pharmaceutical Inc. decided to acquire the Duchenne muscular dystrophy play to get what could become the California company's largest drug. BioMarin believes...
07:00 , Oct 16, 2014 |  BC Innovations  |  Distillery Therapeutics

Indication: Endocrine/metabolic disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Endocrine/metabolic disease Mucopolysaccharidosis a-l-iduronidase (IDUA) Feline studies suggest liver-directed IDUA gene therapy could help treat mucopolysaccharidosis I, which is caused by IDUA deficiency and characterized by...
07:00 , Sep 18, 2014 |  BC Innovations  |  Distillery Therapeutics

Indication: Endocrine/metabolic disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Endocrine/metabolic disease Mucopolysaccharidosis a-l-iduronidase (IDUA) Feline studies suggest intrathecal IDUA gene therapy could help treat mucopolysaccharidosis I (MPS I), which is caused by a deficiency in...
07:00 , Jun 16, 2014 |  BioCentury  |  Regulation

Signal crossing

FDA...
07:00 , Mar 31, 2014 |  BioCentury  |  Regulation

Viral crossroads

Now that the family of seven-year-old Josh Hardy has successfully obtained access to an experimental drug, aided by an explosion of social media support, lawmakers, industry and patients still are left grappling with the fundamental...
08:00 , Feb 20, 2014 |  BC Innovations  |  Distillery Therapeutics

Indication: Endocrine/metabolic disease

Indication Target/marker/pathway Summary Licensing status Publication and contact information Endocrine/metabolic disease Mucopolysaccharidosis a-l-iduronidase (IDUA) In vitro and mouse studies suggest megakaryocyte-targeted gene therapy could help treat mucopolysaccharidosis I (MPS I; Hurler syndrome). In a human...
08:00 , Jan 28, 2013 |  BioCentury  |  Tools & Techniques

BioMarin's brainy buy

The winding down of a 2011 development deal between Zacharon Inc. and Pfizer Inc. gave erstwhile suitor BioMarin Pharmaceutical Inc. a new opening to buy the company. The acquisition is also the latest in a...
08:00 , Nov 5, 2012 |  BioCentury  |  Strategy

Orphanage in Asia

When Alnylam Pharmaceuticals Inc. decided to find an Asian partner for its lead "5x15" RNAi program, Sanofi 's Genzyme Corp. unit emerged as the best option due to its Orphan network in Japan and Sanofi's...