RM004

RM004 is an autologous, ex vivo gene-edited hematopoietic stem and progenitor cell (HSPC) therapy developed by Reforgene Medicine for the treatment of transfusion-dependent alpha-thalassemia, specifically Hemoglobin H-Constant Spring (HbH-CS). The therapy utilizes a Cytosine Base Editor (CBE) to precisely correct the single-base mutation (c.427T>C) in the Hemoglobin Subunit Alpha 2 (HBA2) gene. By restoring the expression of the wild-type alpha-globin chain, RM004 aims to eliminate the need for regular red blood cell transfusions. Clinical data from a first-in-human study (NCT06107400) demonstrated successful engraftment and transfusion independence in treated patients, with significant reductions in HbH and Hb-CS levels and a corresponding increase in Hemoglobin A1.