Expert Interview
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: Duke University
- Associate Professor of Medicine and Pediatrics and Director of the Adult Sickle Cell Program at Duke University.
- Currently cares for 200 patients with Sickle Cell Disease (100 children & 100 adults) and treats 3 adults and 3 children with transfusion dependent thalassemia.
- Research has focused on the epidemiology, risk factors, and prevention of the pulmonary and central nervous system complications of sickle cell disease and includes retrospective and prospective cohort studies and clinical trials; local site co-investigator on trials for GBT-440 and rivapansel.
What were your thoughts on the data presented at ASH on exa-cel?
Added By: c_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: usercc9c06a1What 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: user20c9c0b5What 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: user20c9c0b5Roughly how many patients with sickle cell disease do you currently manage? With Beta Thalassemia?
Added By: c_adminAre You Interested In These Questions?
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