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DURHAM, N.C. & SEATTLE--(BUSINESS WIRE)--May 22, 2023--Last week, at the 26th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), leading epigenetic editing company Tune Therapeutics showcased the power and potential of its TEMPO ^TM genetic tuning platform, by presenting data showing the stable repression of the PCSK9 gene and the effective reduction of LDL-cholesterol levels in non-human primates (NHPs). This data marks a significant waypoint on the path to epigenetic medicine, in that it represents the first demonstration of durable epigenetic gene regulation, in a large animal model, following transient delivery of an epi-editor.

A common therapeutic target for the prevention of cardiovascular disease, it is well established that repressing the PCSK9 gene reduces elevated LDL-cholesterol levels, in turn associated with the formation of artery-clogging plaques. The non-human primate data presented at the ASGCT conference showed that epigenome editing (or genetic tuning) can drive the stable repression of PCSK9 (ongoing past four months) following a single treatment, and without cutting or nicking the DNA, or altering its coding sequence in any way.