Ultragenyx Pharmaceutical Inc.
http://www.ultragenyx.com/
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Latest From Ultragenyx Pharmaceutical Inc.
Bespoke Gene Therapy ‘Playbook’ Outlines Platform Approach To AAV-Based Treatments
Supporting ‘n of 1’ gene therapy development, the playbook from a Foundation for the National Institutes of Health-backed consortium aims to help investigators who are not traditional product sponsors prepare for meetings with US FDA and IND submissions.
EMA Grants First Two PRIME Designations In 2024 To Ultragenyx & Sangamo
Ultragenyx’s antisense oligonucleotide for treating Angelman syndrome and Sangamo’s gene therapy for Fabry disease are the latest products to make it onto the European Medicines Agency’s priority medicines scheme.
From Whiskey To Cancer Drugs: George Nakayama’s Unusual Journey
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Unpartnered Assets To Watch In 2024
Licensing serves as a strategic R&D avenue for major developers amid a shrinking M&A market. In Vivo has detected five high-value unpartnered assets that might be the focus of dealmaking activity in the coming months.
Company Information
- Industry
- Biotechnology
- Pharmaceuticals
-
Biotechnology
- Gene Therapy, Cell Therapy
-
Large Molecule
- Antibodies
- Other Names / Subsidiaries
-
- Dimension Therapeutics
- GeneTx Biotherapeutics LLC
- Ultragenyx
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