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uniQure N.V.

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Latest From uniQure N.V.

Pfizer Matches Beqvez Price To Hemgenix In Hemophilia B, Plans Warranty Program

The drug maker said the program would allow for continued reimbursement when patients change insurance plans. A Sangamo-partnered gene therapy for hemophilia A is also in the works.

Approvals Gene Therapy

US FDA’s April Outlook: Decisions Pending For Rare Pediatric Diseases, New Antibiotics

User fee goal dates in April include four novel agents with breakthrough therapy designations, including Pfizer’s hemophilia B gene therapy and Immunity Bio’s IL-15 superagonist complex for bladder cancer.

Orchard Will Launch Lenmeldy At Just Five Centers

Orchard, which Kyowa Kirin bought in October, gave Lenmeldy a $4.25m list price, exceeding the upper bound of about $3.9m set by ICER.

Approvals Pricing Strategies

CGT Is In Its Infancy For CNS Disorders, But Technical Strides Being Made

Technical challenges and lackluster investor sentiment about the near-term commercial potential of cell and gene therapies in neurological disorders may be holding the field back.

Innovation Market Intelligence
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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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