RG2833

RG2833 (also known as RGFP109) is a brain-permeable small molecule inhibitor of histone deacetylase 1 and 3 (HDAC1/HDAC3), with IC50 values of approximately 60 nM for HDAC1 and 50 nM for HDAC3. It acts by inhibiting the deacetylation activity of these enzymes, leading to increased acetylation of histones and upregulation of target genes such as frataxin. This mechanism is relevant in diseases like Friedreich's ataxia where frataxin deficiency plays a role. Preclinical studies have shown that treatment with RG2833 increases frataxin mRNA and protein levels in neuronal cells and blood from patients. The drug has been granted orphan drug status in both the US and EU for Friedreich's ataxia and has undergone phase I clinical trials for this indication[1][5][7]. There is also preclinical investigation into its use for diffuse intrinsic pontine glioma[4]. The compound was co-developed by The Scripps Research Institute and Repligen Corporation[7].

Other names

RGFP-109RGFP109RGFP 109Benzamide N-[6-[(2-aminophenyl)amino]-6-oxohexyl]-4-methyl-RGFA