Strategic Transformation 2023
Sickle Cell Disease (SCD) is an Inherited Life-Threatening
Hematological Disorder Manifesting Shortly After Birth
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SICKLE CELL DISEASE is a genetic blood disorder caused
by mutations in the HBB gene that causes sickling of RBCs;
this leads to anemia, hemolysis, and VOEs ¹,2
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UPREGULATION OF FETAL HEMOGLOBIN (HbF) is a
naturally validated therapeutic strategy to control
complications of SCD
SCD AFFECTS 3,4,5
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EDITAS EDITS THE HBG1 AND HBG2 PROMOTERS USING ASCAS12a ENZYME, AN APPROACH THAT IS
DESIGNED TO:
Upregulate HbF robustly
Correct anemia with superior red blood cell production and health vs. BCL11A approach
Reduce risk of off-target editing with high fidelity and high efficiency proprietary AsCas12a enzyme
to
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PEOPLE
IN THE U.S.
Reni-cel is potentially a “best in class” medicine with consistent correction of anemia
HBB, B-globin gene; RBC, red blood cell; SCD, sickle cell disease; VOE, vaso-occlusive event.
1. Kato GJ et al. Nat Rev Dis Primers 2018; 4: 18010. 2. Williams TN et al. Annu Rev Genomics Hum Genet 2018; 19: 113-147. 3. Sickle Cell Disorders. Available at: https://www.thelancet.com/pb-
editas assets/Lancet/gbd/summaries/diseases/sickle-cell-disorders.pdf. Accessed June 2023. 4. Wastnedge E et al. J Glob Health 2018; 8 (2): 021103. 5. Sickle Cell Disease. Available at:
https://www.nhlbi.nih.gov/health/sickle-cell-disease. Accessed June 2023.
MEDICINE
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