Expert Interview
A Second View: Discussing the potential Vertex & CRISPR's exa-cel in patients with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT)
Ticker(s): VRTX, CRSPInstitution: Tulane University
- Professor of Medicine, Pediatrics & Pathology, Chief of Hematology & Medical Oncology, & Director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University School of Medicine.
- Currently manages 200 patients with hemophilia A and B and 60 patients with sickle cell disease.
- Research focuses on coagulation disorders; oversees an active research program that receives funding from the National Institutes of Health as well as numerous other federal and industry sources.
What were your thoughts on the data presented at ASH on exa-cel?
Added By: max_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: max_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: max_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: max_adminRoughly how many patients with sickle cell disease do you currently manage? With Beta Thalassemia?
Added By: max_adminAre You Interested In These Questions?
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