ASH 2023 | PB-04 in beta thalassemia intermedia and SCD
Kevin Kuo, MD, MSc, FRCPC, University Health Network, Toronto, Canada, introduces PB-04, an oral fetal globin stimulant which suppresses four repressors of the fetal hemoglobin gene promotor (BCL-11A, LSD-1, HDAC-3, KLF-1). Dr Kuo highlights some initial results from a Phase Ib trial (NCT04432623) of PB-04 in sickle cell disease (SCD) and beta thalassemia intermedia, which reports fetal hemoglobin induction, additive activity with hydroxyurea and improved red blood cell sickling parameters. This interview took place at the 65th ASH Annual Meeting and Exposition, held in San Diego, CA.
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Disclosures
Consultancy: Agios Pharmaceuticals, Alexion Pharmaceuticals, Bristol Myers Squibb, Forma Therapeutics, Pfizer, Novo/Nordisk, Vertex Pharmaceuticals
Research Funding: Agios Pharmaceuticals
Honoraria: Bristol Myers Squibb, Novo/Nordisk
Board of Directors/Advisory Committee: Bioverativ, Sanofi, Sangamo
