Expert Interview
A Second Look: Discussing Fulcrum's pociredir and other oral HbF inducers for sickle cell disease (SCD)
Ticker(s): FULCInstitution: Emory University
- Associate professor of pediatrics at Emory University.
- Currently manages ~150 patients with Sickle Cell Disease.
- Research focuses on the mechanisms of globin switching and the pharmacogenomics of hydroxyurea in SCD.
Of the different mechanisms being investigated, which of them seems to have the most promise moving forward?
Added By: max_adminCan you provide a broad overview of fetal hemoglobin (HbF) inducers and pyruvate kinase activators in development for sickle cell disease (SCD), including their mechanisms and available clinical data?
Added By: max_adminWhat are your thoughts on the efficacy of Fulcrum’s pociredir, particularly regarding its HbF increases at different dosage levels, and what level of increase would be needed to reduce fatigue and vaso-occlusive crises (VOCs)?
Added By: max_adminWhat are the safety concerns associated with HbF inducers like pociredir, particularly regarding potential cancer risks and other adverse events such as neutropenia and GI toxicity?
Added By: max_adminHow do you compare the potential of HbF inducers versus pyruvate kinase activators for treating SCD, and do you foresee one class being more successful than the other?
Added By: max_adminRegarding the kinetics of response, do you expect HbF induction to plateau within the 12-week treatment period, and could there be compensatory mechanisms that might reduce long-term efficacy?
Added By: max_adminAre You Interested In These Questions?
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