Expert InterviewVRTX
Slingshot members are talking to an expert! The topic is:
Discussing the potential Vertex & CRISPR's exa-cel in patients with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT)
Executed on:Jan 26, 2023 12:00 PM EDT
Call Duration:30 minutes
Expert Profile
ExpertBlinded Expert
Institution: 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.
Interview Goal
On this call, we aim to discuss exagamglogene autotemcel (exa-cel) and the Phase 3 studies of exa-cel in patients with TDT(CLIMB THAL-111) and in patients with severe SCD (CLIMB SCD-121), which were presented at ASH 2022.