Ultragenyx Pharmaceutical Inc.
http://www.ultragenyx.com/
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Latest From Ultragenyx Pharmaceutical Inc.
Gene Therapy Biotechs Treating Sanfilippo Syndrome Hit Cash Crunch
Despite promising clinical data and solid regulatory plans, biotech firms hoping to address the unmet need in Sanfilippo Syndrome through gene therapy are facing financial struggles.
Orphalan Flies Solo In US After Wilson’s Disease Drug Win
Naseem Amin, the privately owned group’s CEO, tells Scrip that, just as in Europe, Orphalan has the resources to commercialize Cuvrior in the US: “We understand the market and we don't need external partners."
Bayer Consolidates Next-Generation Gene Editing Focus With Mammoth Deal
The German major will collaborate with the US biotech to develop CRISPR gene-editing therapies, solidifying its interest in next-generation therapeutics.
Deal Watch: J&J Begins 2022 With Trio Of Technology Alliances
Heading into the J.P. Morgan Healthcare conference, pharmas have been focusing on discovery alliances. J&J’s deals look into novel tissue-targeted drugs, ionic liquid delivery technology and cancer vaccine R&D. Lilly teams with Entos on novel methods for delivering neurology drugs.
Company Information
- Industry
- Biotechnology
- Pharmaceuticals
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Biotechnology
- Gene Therapy, Cell Therapy
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Large Molecule
- Antibodies
- Other Names / Subsidiaries
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- Ultragenyx
- Dimension Therapeutics