Novel DUSP1 and FLT3-JAK2-IKK1 Degraders for Treating Relapsed and Recurrent Leukemia
Summary
Next-generation orally bioavailable degrader programs targeting DUSP1 and NFKB1 suppress JAK-STAT and NFκB signaling to restore treatment sensitivity in relapsed and refractory leukemia and potentially other cancers.
Overview
Cincinnati Children’s has developed novel, orally bioavailable PROTAC degraders targeting DUSP1 and NFκB pathway regulators (FLT3–JAK2–IKK1 axis) to overcome treatment resistance in leukemia. These agents address a large, high-unmet-need relapsed/refractory (R/R) population, with early preclinical validation demonstrating reversal of resistance to TKIs and immunotherapy across multiple hematologic malignancies.
Applications
Leukemia and Cancer
Value Proposition
Multi indication platform, Combination-enabling, High unmet need, Orphan designation potential, Expansion into solid tumor indications
Market Overview
10-15 B TAM around leukemia space
Investigator
Mohammad Azam, PhD



