FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Demonstrates Proof of Mechanism and Proof of Concept after a Single Dose and after Multiple Daily Doses in a Phase 1 Study of Patients with Sickle Cell Disease.

R. Clark Brown, Kimberly Cruz, Theodosia A. Kalfa, Frans A. Kuypers, Santosh L. Saraf, Jeremie H. Estepp, Luke R. Smart, Punam Malik, Mark Lerman, Renae Mayer, Maria D. Ribadeneira, Sanjeev Forsyth, Patricia Schroeder, Eric Wu, Patrick Kelly, Marilyn J. Telen. FT-4202, an Allosteric Activator of Pyruvate Kinase-R, Demonstrates Proof of Mechanism and Proof of Concept after a Single Dose and after Multiple Daily Doses in a Phase 1 Study of Patients with Sickle Cell Disease. Blood. 2020 Nov 5; 136(Supplement 1):19-20.