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Investor Presentaiton

Target protein Molecular glue molecule E3 ligase Targeting the previously undruggable: A novel CELMOD for Sickle Cell Disease Transformational potential Oral small molecule that increases fetal hemoglobin to functionally cure sickle cell anemia (e.g., eliminate pain crisis, prevent long term organ damage). Causal human biology Genetically validated targets that lead to persistence of fetal hemoglobin (HbF) are associated with improved clinical outcomes in patients with sickle cell anemia. Beta-globin locus 5 4 3 2 1 ε Gy Ay δ Β HbS Matching modality to mechanism Through our CELMOD proteomics initiative, we have identified CELMODs that degrade HbF genetic targets and increase HbF in pre-clinical models. HbF expression 8000- wwww 6000- Vehicle control CELMOD (high) CELMOD (low) 4000- 2000- TH Path to clinical proof-of-concept α B disease 10% HbF 20% 30% Hydroxyurea a LCR HSS Embryonic Fetal Adult 3'HS1 HbF Ill Bristol Myers Squibb™ healthy Reduced Reduced mortality recurring events Asymptomatic presentation Not for Product Promotional Use 37
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