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Ultragenyx Pharmaceutical Inc.

http://www.ultragenyx.com/

Latest From Ultragenyx Pharmaceutical Inc.

With Hopeful Interim Data, Ultragenyx Bets On GeneTx, Angelman Syndrome Candidate

Ultragenyx is buying its Angelman syndrome partner GeneTx for $75m on the heels of an interim Phase I/II readout that shows signs of clinical activity and appears to resolve earlier safety worries.

Rare Diseases Business Strategies

Sanofi/Sobi’s Next-Gen Factor VIII Therapy BLA Heats Up Biotech-Driven Hemophilia Arms Race

Sanofi/Sobi’s efanesoctocog is in line for US FDA action three months after CSL/uniQure’s gene therapy user fee goal, while BioMarin’s Roctavian resubmission and Novo Nordisk’s concizumab are waiting in the wings.

Gene Therapy Drug Review

Bayer Sells Nebido To Grünenthal Amid Shift In Focus To Cell And Gene Pipeline

The major will use funds from the sale of its male hypogonadism product to invest in future innovation, which is likely to focus on high-risk high-reward cell and gene drug candidates.

Deals Companies

Finance Watch: Theravance Eliminates Debt In $1.1bn Trelegy Royalty Sell-Off

Public Company Financings: Theravance sold its 85% interest in Trelegy royalties from GSK and Innoviva sold its 15% interest for up to $1.8bn in total. Also, ProKidney grossed $597m from its SPAC merger, Pliant raised $200m in a post-Phase IIa offering and Marinus earned $110m in a PRV sale.

Financing Business Strategies
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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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