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Expert Interview

Slingshot members are talking to an expert! The topic is:

Another View: Exploring gene therapy treatments in the hemophilia space including those from BioMarin and Spark Therapeutics

Ticker(s): BMRN, ONCE, PFE

Who's the expert?

Institution: University of Kentucky

  • Director, Hemophilia Treatment Center at the University of Kentucky & Associate Professor of Medicine.
  • Currently manages 50 patients with hemophilia A and 12 patients with hemophilia B.
  • Familiar with gene therapy research for hemophilia and involved in a Factor IX trial with St. Jude.

Interview Goal
To discuss the following gene therapies currently in development including:
  • 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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