PFE-001 / Pfizer - LARVOL DELTA

[VIRTUAL] IDENTIFICATION OF NON-COVALENT MODIFIERS OF HB S WITH A NOVEL HEMOGLOBIN BINDING SITE (EHA 2020) - "Recently, it was shown that a non-covalent small molecule, PFE-001, binds to the α-chains of Hb S, and demonstrates efficacy in the Townes mouse model of SCD...Conclusion These results indicate that the a-chain is not the only binding site available for stabilization of the oxygenated state of hemoglobin and suggest that targeting the b-chain binding site has potential therapeutic value. Further, these results confirm the ability of non-covalent small molecules to impact disease pathology, expanding the possibilities for hemoglobin modification as a treatment for Sickle Cell Disease."

Cardiovascular • Complement-mediated Rare Disorders • Gene Therapies • Genetic Disorders • Hematological Disorders • Pain • Sickle Cell Disease