Epsilen Bio’s research is based on a novel modality of gene silencing that exploits epigenetics to convey inheritable states of transcriptional repression at desired target genes.
This strategy is based on transient delivery of combinations of Engineered Transcriptional Repressors (ETRs), chimeric proteins containing a programmable DNA binding domain fused to one or more epigenetic effector domains. Once delivered into the cell, the ETRs bind to – and synergize at – their intended target locus to promote its transcriptional repression via de novo deposition of DNA methylation, an epigenetic mark known to be permanently inherited throughout cell division.
The ETR technology (or « epi-silencing ») can be applied to both in vivo and ex vivo approaches, and can promote high levels of long-term stable gene silencing – even when the gene is present in multiple copies or for multiple genes at the time («multiplexing»). Further advantages of the epi-silencing technology include: (i) it is highly specific, (ii) it does not induce DNA breaks, and (iii) it can be reverted at will by administration of clinically approved agents. Overall, the hit-and-run nature of this platform coupled with the long-term stability of the induced epigenetic modifications makes epi-silencing an attractive solution for the treatment of a number of debilitating disease conditions.