Core Viewpoint - Oryzon Genomics has initiated the RESTORE clinical trial for iadademstat, targeting sickle cell disease (SCD) with the aim of increasing fetal hemoglobin (HbF) levels, which is recognized as a significant clinical endpoint by the FDA [1][3][6] Company Overview - Oryzon Genomics, founded in 2000 in Barcelona, Spain, is a clinical-stage biopharmaceutical company specializing in epigenetics and personalized medicine, particularly in CNS disorders and oncology [4] - The company has a clinical portfolio that includes two LSD1 inhibitors: vafidemstat (Phase III-ready) and iadademstat (Phase I/II) [4] Clinical Development - The RESTORE trial is a multi-center, open-label Phase Ib study designed to enroll approximately 40 adult patients with SCD to evaluate the safety, tolerability, and recommended Phase 2 dose of iadademstat [1][2] - Iadademstat is an oral, selective LSD1 inhibitor that aims to re-induce HbF through epigenetic reprogramming, a mechanism similar to certain FDA-approved gene therapies for SCD [2][5] - The drug has shown good tolerability in previous studies, with nearly 200 patients treated, supporting confidence in its safety profile as it enters the RESTORE trial [2][6] Market Need - Sickle cell disease is the most common inherited blood disorder in the U.S., representing a significant unmet medical need with limited treatment options [3] - By increasing HbF levels, iadademstat aims to mitigate key complications of SCD, such as vaso-occlusion and organ damage, which are critical factors affecting morbidity and survival [3]