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Pediatric Acute Myeloid Leukemia: Accelerating the discovery of low-toxicity drug candidates

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IN PARTNERSHIP WITH THE US DEPARTMENT OF DEFENSE, THE MINISTRY OF INNOVATION OF QUEBEC, AND MCGILL UNIVERSITY

Targeting fusion oncoproteins is key to advancing pediatric AML treatment. 

Currently, AML therapies exhibit high toxicity and limited efficacy, resulting in a 5-year survival rate of only 60%. This project is dedicated to developing therapies that specifically target fusion oncoproteins, the primary drivers of AML.

We have developed candidates selectively targeting 3 of the most common oncotargets driving pediatric AML. These compounds exhibit lead-like efficacy, with IC50 values in the mid to low nM range, and have favorable low toxicity profiles.