Fulcrum Therapeutics Inc. has released a corporate presentation detailing its strategic focus on developing oral small molecules aimed at modifying gene expression in rare diseases, particularly in the field of benign hematology. The presentation highlights Pociredir, a potential best-in-class oral small molecule for inducing fetal hemoglobin (HbF) in sickle cell disease $(SCD)$, which has received Fast Track and Orphan Drug Designations. Data disclosure for Phase 1b PIONEER trial cohorts is planned for early Q3 2025 and year-end 2025. Additionally, the company plans to submit an Investigational New Drug $(IND.AU)$ application in Q4 2025 and reports a cash position of $226.6 million as of March 31, 2025, providing a financial runway into at least 2027. You can access the full presentation through the link below.
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