- 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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Another View: Exploring gene therapy treatments in the hemophilia space including those from BioMarin and Spark TherapeuticsTicker(s): BMRN, ONCE, PFE
Name: Dr Vlad Radulescu - MD
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.
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