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Latest From uniQure N.V.

No AdComm Puts Sarepta On Path To Blockbuster Success With Duchenne Gene Therapy

The US FDA’s waiving of an advisory committee is another promising signal that Sarepta's Duchenne gene therapy SRP-9001 could be approved on 29 May and set it up to become a bigger earner than Novartis’s Zolgensma.

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CSL/UniQure’s Hemgenix To Enter Uncertain EU Market After Regulatory Nod

The firms’ one-time hemophilia B gene therapy has been approved in the EU just months after a US nod, but concerns remain about its prospects on a market that has seen several setbacks for other gene therapies.

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Deal Watch: BMS Returning IL-12 Candidate To Frequent Partner Dragonfly

The firms’ partnership goes back to pre-merger Celgene, and BMS still has Dragonfly candidates in its pipeline. Ji Xing gets hypertension candidate from Phase Bio.

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Bountiful Approvals May Be Ahead As FDA Starts 2023 With Almost 60 Novel Agents Under Review

The upcoming year could see a rebound approvals if the bigger-than-usual queue of novel agents with 2023 user fee goal dates pans out.

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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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