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ASH 2023 | Investigating AB1 as a tool to upregulate fetal hemoglobin in SCD: preclinical and clinical findings

Nirmish Shah, MD, Duke University, Durham, NC, discusses the preclinical and clinical findings regarding the use of AB1, a DNMT1 protein depleter, as an agent to upregulate fetal hemoglobin in sickle cell disease (SCD). The drug was tested as a proof of concept in murine models with and without the addition of hydroxyurea, before being moved on to the first clinical trial of AB1 in SCD (NCT05261711). To date, AB1 has been found to be safe at low doses during the ongoing dose-escalation phase. This interview took place at the 65th ASH Annual Meeting and Exposition, held in San Diego, CA.

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Disclosures

Advisory board: Agios, Bluebird Bio, CSL Behring, Emmaus, Forma Therapeutics, Novo Nordisk, Pfizer, Vertex
Research Funding: Pfizer
Speakers Bureau: Pfizer, Alexion, Novartis