SCIENCE Foundational Science
Epigenetics is nature’s fundamental mechanism for gene control and cell differentiation. Through the pioneering research of our founding scientific advisors and our own work, we have developed a deep understanding of gene regulation, genomic architecture and epigenetic mechanisms, which guides our approach to creating a new class of medicines to treat or cure diseases.
Epigenetic Regulation of Genomic Architecture
Gene expression is regulated by the dynamic 3-dimensional structure of DNA. Together with scaffolding elements, the shape of DNA regulates access to specific genes and their regulatory elements. Reversible modifications on top of DNA or its scaffolding, known as epigenetic modification, control the winding or formation of DNA loops without altering the underlying sequences. These evolutionarily conserved loops of chromatin, called insulated genomic domains (IGDs), act as nature’s innate control system for gene expression.
IGDs Structure and Function
IGDs are the fundamental structural and functional units of gene regulation that control the entire life cycle of every cell type in the body. We have identified approximately 15,000 IGDs across 23 chromosomes in every cell, each containing one or multiple genes and their associated regulatory elements. Anchored by CTCF and cohesin at the base of each loop, IGDs are insulated from outside transcription. Importantly, data have shown clear links between dysfunctional IGDs and diseases.
IGDs and Disease
Irrespective of etiology, most diseases manifest due to dysregulated gene expression occurring because of changes to IGDs. Since our founding, we have developed a comprehensive understanding of IGD biology and the specific regulatory sequences within IGDs called Epigenomic Zip codes or “EpiZips” that represent unique targets for drug development.
Our understanding of IGDs and EpiZips is a core pillar of our platform. Using our technology, we create programmable mRNA medicines designed to make specific epigenetic changes and correct abnormal gene expression to treat or cure diseases.