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Expert Interview

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

Examining the market size for Hemophilia B gene therapy treatments with a focus on uniQure & Spark

Ticker(s): QURE, ONCE, PFE

Who's the expert?

Institution: Mount Sinai

  • Associate Professor & Director of the Hemophilia Program in the Division of Hematology and Medical Oncology at the Icahn School of Medicine at Mount Sinai, Regional Director of ATHN funded Hemophilia Treatment Centers in New England.
  • Currently manages 120 patients with Hemophilia B.
  • Research focuses on gene therapy of blood diseases. Pioneered the research into Adeno-Associated Virus (AAV) and its first use as a gene therapy vector; early work culminated in the first clinical trial in for gene therapy of patients with Fanconi Anemia Group C and Group A.

Interview Goal
To better understand the market size for gene therapy treatment of Hemophilia B including QURE's AMT-061 and ONCE's Fidanacogene elaparvovec (SPK-9001).

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