Epigenetic biologics of a new kind

Any cell in an organism includes all genes of that organism's genome. The difference in structure and function of cells results from epigenetic silencing of 'unnecessary genes' which occurs in great part by DNA methylation. Taking control of the epigenome is a novel and exciting biomedical challenge. Moreover, in disease the aberrant DNA methylation in key regulatory areas (e.g. promoters) can inhibit gene transcription. DNA methylation abnormalities are increasingly found in numerous pathologies, however we usually cannot say if these abnormalities are in fact causative for the disease. This field enables novel therapeutic and experimental approaches via epigenetic mechanisms, however a number of challenges have been preventing novel therapeutics of this kind.

We develop custom-designed molecules comprised of putative DNA demethylases fused with sequence-specific DNA binding domains and use these tools to re-activate or enhance the silenced intrinsic genes in cells. In contrast to existing techniques, targeted demethylation opens a specific gene promoter to adaptive, context-appropriate activation in situ, and offers superior potential to future therapeutic regulatory tactics, not only in epigenetically-mediated disease mechanisms but more broadly where gene function enhancement is desirable. Epigenter develops protein-based 'biologics' that enable epigenetic editing in vivo without the need for transgenesis or complex packaging tools.

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