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Exploring gene therapy treatments in the hemophilia space including those from BioMarin, Spark Therapeutics, and UniQure

Ticker(s): BMRN, ONCE, QURE

Who's the expert?

A hematologist with significant experience treating patients with Hemophilia.

Interview Goal
Thinking ahead of first gene therapy approval/launch, we'll dig into the hemophilia landscape and cover
  • Valoctocogene Roxaparvovec (BMN 270): gene therapy designed for the treatment of Hemophilia A in development by BioMarin.
  • SPK-8011: for Hemophilia A
  • Fidanacogene elaparvovec, previously SPK-9001: an investigational bio-engineered AAV vector utilizing a high-activity factor IX transgene currently in development by Spark Therapeutics/Pfizer.

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