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Ultragenyx Pharmaceutical Inc.

http://www.ultragenyx.com/

Latest From Ultragenyx Pharmaceutical Inc.

Kyowa Kirin’s North America Expansion Progresses With Crysvita Commercial Takeover

The Japanese pharma has taken over commercialization of the rare disease drug from Ultragenyx under a planned transition and as part of a broader growth strategy in North America.

North America Business Strategies

Product Quality ‘Wild Card’? Sarepta’s Gene Therapy Manufacturing Process Change Means More Empty Capsids

SRP-9001 intended for commercialization is not analytically comparable to the product used in earlier clinical studies; FDA CMC reviewer said agency does not mandate the extent of empty or full capsids but, instead, looks at the totality of data to assess safety and efficacy.

Advisory Committees Gene Therapy

Deal Watch: Dianthus Will Reach Public Markets Via Reverse Merger With Stalled-Out Magenta

After shutting down its trial for a leukemia conditioning agent due to a patient death, Magenta’s review of strategic alternatives concludes with a combination with complement inhibitor-focused Dianthus.

Deal Watch Business Strategies

Supplemental Filings: Pink Sheet Notebook On Disease Lobbying, Dan Leonard’s Next Act, And More

Quick takes on Ultragenyx’s CEO lobbing the US FDA for rare disease policy changes, the agency naming a new deputy assistant commissioner for media affairs, the former CEO of AAM starting a consulting firm, and more.

Leadership Rare Diseases
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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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