Drug intelligence / Profile preview

arginine butyrate + epoetin alfa

Development stage
Unknown
Lead developer
Boston University
Modality
Recombinant Proteins and Enzymes, Small Molecules
Administration
Intravenous, Subcutaneous
01

Overview

Arginine butyrate in combination with epoetin alfa is an experimental therapeutic regimen developed by Boston University for the treatment of beta-thalassemia intermedia. Arginine butyrate is a small molecule short-chain fatty acid derivative that acts as a fetal globin gene inducer, working primarily by inhibiting histone deacetylation to stimulate gamma-globin chain production. Epoetin alfa (also known as recombinant human erythropoietin or EPO) is a biologic hematopoietic growth factor that prolongs erythroid cell survival and stimulates red blood cell production. The combination therapy was evaluated in Phase II clinical trials to determine whether the two agents could produce additive hematologic responses by increasing fetal hemoglobin levels and enhancing erythropoiesis in patients with thalassemia intermedia, particularly those with relatively low baseline endogenous EPO levels.

Other names
arginine butyrate and epoetin alfaarginine butyrate plus epoetin alfa
02

Targets

HDAC (HDAC family)Erythropoietin Receptor

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