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Expert Interview

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

Exploring the recent hemophilia A - SB-525 gene therapy data from Sangamo

Ticker(s): SGMO, PFE

Who's the expert?

A hematologist with experience fn mainstream therapies in hemophilia A and knowledge of SB-525.

Interview Questions
Q1.

Please tell us about your clinical experience. How many patients do you currently treat with hemophilia A? What percent of them are in severe condition, and what are the best treatment options available to increase FVIII activity?

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Q2.

Can you explain to us the mechanism of action of SB-525 liver-targeted recombinant adeno-associated virus vector carrying B-domain deleted F8 gene?

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Q3.

According to the data, the first 2 patients treated at the 3e13 vg/kg dose level achieved normal sustained factor VIII levels with no reported bleeding events and no factor usage during the 24 weeks of follow-up. How does this compare, and what is your take on the drug’s efficacy so far?

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Q4.

  1. One patient experienced hypotension(grade 3) and fever(grade 2) 6 hours after completion of SB-525 infusion.
  2. In the 3e13 vg/kg cohort two subjects experienced a transient grade 1 alanine aminotransferase elevation (>1.5 x baseline) managed with a tapering course of oral steroids. Neither resulted in a loss of FVIII activity levels
How common is this in Hemophilia A treatments? Any reason to worry about these adverse events?

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Q5.

How much of a need is there for a safer/more efficient treatment option in severe hemophilia A?

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Q6.

Is the single intravenous infusion an important benefit to your patients? Also, no bleeding events have been observed in any of the 4 subjects treated at the 3e13 vg/kg dose. How does it compare to other therapies?

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Q7.

Based on the accumulating results from the Alta study, FDA has granted regenerative medicine advanced therapy designation for SB-525. How important is this aspect?

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Q8.

How likely would you be to prescribe SB-525 in the future?

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