Ultragenyx Pharmaceutical Inc.
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Latest From Ultragenyx Pharmaceutical Inc.
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.
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 extricating Daiichi Sankyo from the Ranbaxy acquisition to enabling the rapid development of blockbuster Enhertu, George Nakayama has had a winding career path through multiple key strategic positions and decisions. He shares his experience and insights, and a good dose of humor, in this exclusive interview.
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.
- Gene Therapy, Cell Therapy
- Other Names / Subsidiaries
- Dimension Therapeutics
- GeneTx Biotherapeutics LLC
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