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Spark Therapeutics, Inc.

https://sparktx.com/

Latest From Spark Therapeutics, Inc.

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.

Approvals Gene Therapy

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.

Deal Watch Business Strategies

Pfizer’s Gene Therapy Asset Chases UniQure/CSL’s Hemgenix After Pivotal Hemophilia B Success

Pfizer’s fidanacogene elaparvovec has hit the mark in a Phase III hemophilia B trial just a month after rival UniQure’s Hemgenix won US approval, with Pfizer’s plans for regulatory action raising the stakes for Hemgenix.

Clinical Trials Blood & Coagulation Disorders

Value-Based Agreements On The Cards For UniQure/CSL’s $3.5m Hemophilia B Gene Therapy

While Hemgenix has courted criticism since its $3.5m price tag was unveiled, a drug pricing expert has told Scrip discounts and value-based agreements could make the therapy more palatable to payers.

Pricing Strategies Blood & Coagulation Disorders
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Company Information

  • Industry
  • Pharmaceuticals
    • Drug Delivery
    • Drug Delivery
      • Macromolecule
      • Site Specific
  • Biotechnology
    • Gene Therapy, Cell Therapy
  • Other Names / Subsidiaries
    • Genable Technologies Limited
    • Roche Holding AG (RHHBY)
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