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

Ticker(s): BMRN, QURE, ONCE

Who's the expert?

Name: Dr Guy Young - MD

Institution: University of Southern California

  • Director of the Hemostasis and Thrombosis Center at Children’s Hospital Los Angeles, Professor of Pediatrics at USC & Chair of the Scientific and Standardization Committee on Factor VIII, Factor IX, and Rare Bleeding Disorders of the International Society on Thrombosis and Haemostasis.
  • Currently manages 320 patients with hemophilia A and B
  • Research activities encompass clinical trials in hemophilia, the use of novel anticoagulants in children, and the development of the global hemostasis assays to monitor the effects of medications used to treat bleeding in 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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