Ultragenyx Pharmaceutical Inc.
http://www.ultragenyx.com/
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Latest From Ultragenyx Pharmaceutical Inc.
Finance Watch: SPACs Attempt A Comeback Ahead Of Full IPO Recovery
Public Company Edition: Several special purpose acquisition corporations have emerged, including various health care- and life science-focused SPACs. Also, Alvotech entered into a $965m refinancing agreement and Avidity grossed $461m in a follow-on offering, among other updates.
Ultragenyx Data De-Risk Phase III Trial Of Setrusumab
The Mereo-partnered drug is in pivotal development for osteogenesis imperfecta. Ultragenyx also recently reported positive Phase III data for a rare disease gene therapy.
Biogen, Ionis End ALS Program; Ionis Will Advance Angelman Asset Alone
Development has ended for BIIB105 in ALS after it failed to clear a crucial biomarker and show clinical benefit. Also, Biogen opted not to advance ION582 for Angelman syndrome, so Ionis will go it alone.
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.
Company Information
- Industry
- Biotechnology
- Pharmaceuticals
-
Biotechnology
- Gene Therapy, Cell Therapy
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Large Molecule
- Antibodies
- Other Names / Subsidiaries
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- Dimension Therapeutics
- GeneTx Biotherapeutics LLC
- Ultragenyx
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