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Expert Interview

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

Reviewing EDIT-301 as an experimental cell therapy medicine for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT)

Ticker(s): EDIT, VRTX, BLUE, CRSP

Who's the expert?

Institution: Howard University

  • Professor of Medicine and Microbiology/Immunology & Director or the Center for Sickle Cell Disease at Howard University (previously held positions at the NIH and Johns Hopkins).
  • Manages 355 patients with Sickle Cell Disease, 2 active Beta-Thalassemia patients and 1 HbH disease patient.
  • Research interests include the use of genetic tools to understand and treat sickle cell disease and complications of transfusion therapy.

Interview Questions
Q1.

Please describe your background and clinical setting

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Q2.

How many patients with sickle cell disease do you manage?

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Q3.

What is the current standard of care for sickle cell disease?

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Q4.

How well managed are patients with sickle cell disease?

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Q5.

What are you overall thoughts on the potential of EDIT-301?

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Q6.

What were your impressions of the recent data readout from the RUBY clinical trial?

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Q7.

Where would EDIT-301 fit into your treatment algorithm if it was approved?

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Q8.

Can you rank your excitement for EDIT-301 on a scale of 1-10?

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