The life-saving potency of the SNIM® RNA technology platform has been demonstrated in a recent preclinical study in a mouse model of the fatal human hereditary disease surfactant protein B deficiency. The results of the study have been published in Nature Biotechnology on 09 January 2011 (doi:10.1038/nbt.1733).
SNIM® RNA is stabilized, non-immunogenic messenger RNA. In contrast to conventional mRNA it can be applied repeatedly. In this study, the team headed by Carsten Rudolph has demonstrated that the strong activation of the innate immune system by conventional mRNA can be overcome by replacing 25% of its uridine and cytidine residues with 2-thiouridine and 5-methyl-cytidine, respectively. Doing so abrogates the interaction of the RNA with the Toll-like receptors TLR3, TLR7, TLR8 and with RIG-I. The interaction with these sensors would otherwise elicit inflammatory responses. The modifications described in the recent publication also stabilize the RNA molecule. Therefore, minor doses of SNIM® RNA introduced into cells at low dosing frequency lead to a sustained production of proteins encoded by the SNIM® RNA. A single intramuscular injection of erythropoietin encoding SNIM® RNA significantly raised the hematocrit in mice. Ethris envisages a strong potential of the SNIM® RNA platform in regenerative medicine and in the treatment of metabolic disorders.