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Ultragenyx Pharmaceutical Inc.

http://www.ultragenyx.com/

Latest From Ultragenyx Pharmaceutical Inc.

Regenxbio: On The Cusp Of Becoming A Commercial Gene Therapy Player

Partnered with AbbVie on a wet AMD gene therapy, Regenxbio is also advancing wholly owned candidates for Duchenne muscular dystrophy and MPS I and MPS II.

Gene Therapy Business Strategies

The Real World Is Not Enough: Comparability Is Common Obstacle For RWE In US FDA Decisions

Unsuccessful attempts to use real-world evidence for approvals highlight the need to demonstrate relationship between real-world data and clinical trial patients. The Pink Sheet examines what went wrong for Bluebird/Bristol’s Abecma, Ultragenyx’s Dojolvi, and Karyopharm’s Xpovio.

Real-World Evidence Review Pathway

Gene Therapy For Lethal Glycogen Storage Disease Wins EMA PRIME Designation

Investigational products from Ultragenyx and MinervaX secured a place on the European Medicines Agency’s priority medicines scheme this month.

Research & Development Review Pathway

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
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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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