Expert Interview
An updated look at Vertex & CRISPR's exa-cel in patients with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT)
Ticker(s): VRTX, CRSPInstitution: Boston University
- Professor of Medicine, Pediatrics, Pathology and Laboratory Medicine at Boston University
- Treats 420 patients with sickle cell disease
- Published 460+ articles and 3 textbooks on SCD and has a research focus on disorders of red blood cells
What were your thoughts on the data presented at ASH on exa-cel?
Added By: ben_adminHow many of your pts would you currently prescribe exa-cel for? How are you thinking about the competitive dynamic with Zynteglo? What would cause you to prescribe on over the other?
Added By: ben_adminWhat is the feasibility of reduced toxicity/intensity conditioning (RTC/RIC) to be used with these types of gene therapies? What kind of timeline do you expect for RTC/RIC to come into play?
Added By: ben_adminWhat are the barriers for adoption of these types of therapies (zynteglo, exa-cel)? How much of a barrier are non-direct factors (impact to fertility (chemotherapy), process of stem cell mobilization) vs. product efficacy/safety?
Added By: ben_adminRoughly how many patients with sickle cell disease do you currently manage? With Beta Thalassemia?
Added By: ben_adminAre You Interested In These Questions?
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