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Latest From uniQure N.V.

Pfizer’s Hemophilia A Gene Therapy Superior To Prophylaxis In Phase III

The company’s giroctocogene fitelparvovec, partnered with Sangamo, could compete with BioMarin’s hemophilia A gene therapy Roctavian, but longer-term data may be needed.

Gene Therapy Rare Diseases

uniQure Hopeful In Accelerated Approval Potential For Huntington’s Disease

The biotech announced updated interim Phase I/II data for its gene therapy AMT-130, showing statistically significant improvement compared with an external control.

Gene Therapy Clinical Trials

Stock Watch: Biotechs Sell The Family Silver

When loss-making biotech companies mix debt with sluggish sales, difficult options like restructuring and asset sales remain.

Stock Watch M & A

Getting To Global Is A Hurdle For Cell And Gene Therapies

Only three of the 10 new therapies approved by the US FDA also have been approved in the European Union, while both of the EU gene therapy approvals followed FDA clearance, a Pink Sheet analysis shows.

Regional Comparisons Gene Therapy
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Company Information

  • Industry
  • Pharmaceuticals
    • Drug Delivery
  • Biotechnology
    • Antisense, Oligonucleotides
    • Gene Therapy, Cell Therapy
    • Large Molecule
  • Other Names / Subsidiaries
    • Amsterdam Molecular Therapeutics (AMT)
    • Corlieve Therapeutics SAS
    • InoCard GmbH
    • uniQure Germany GmbH
    • uniQure U.S.A.
    • uniQure The Netherlands
    • uniQure Inc.
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