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Ultragenyx Pharmaceutical Inc.

http://www.ultragenyx.com/

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An analysis of the most valuable research-stage products pinpoints several neurological candidates – and a deeper dive into this segment shows more CNS therapies that look to be worth big money.

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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.

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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.

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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.

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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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