- 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.
Exploring gene therapy treatments in the hemophilia space including those from BioMarin, Spark Therapeutics, and UniQureTicker(s): BMRN, ONCE, QURE
A hematologist with significant experience treating patients with Hemophilia.
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