DIRECTING THE HUMAN
GENOME TO TREAT & CURE DISEASE
WE ARE FOCUSED ON ENABLING A NEW FUTURE OF DISEASE MANAGEMENT BY MAPPING THE TOPOLOGY OF GENOMIC CONTROL, UNDERSTANDING THE BIOLOGY OF DISEASE, AND ENGINEERING PRECISION THERAPEUTICS FOR CLINICAL BENEFIT.
Topology of Genomic Control
Currently, we are generating detailed genomic maps of the physical location, target sequences and characteristics of the GenomStats™, and adding them to our growing proprietary database.
Biology of Disease
We are linking disease biology pathways with the appropriate GenomStats™ for therapeutic intervention, precisely targeting those that control both single-gene and multigene disorders.
We are designing, engineering and manufacturing
Omega Controllers™ to target the GenomStats™ for the cell types of interest, with the ability to downregulate or upregulate genomic activity for therapeutic effect.
AN UPDATED VIEW OF THE CENTRAL DOGMA — ORGANIZATION OF THE DNA AND GENOMIC ACTIVATION
The central dogma defines how we understand the role of the structure and function of the genome in driving biological processes. We have illuminated the critical role of DNA structure in genomic activation — allowing us to intervene upstream of transcription, ultimately resulting in protein creation.
Genomic activity may be tuned, i.e., either upregulated or downregulated depending on biological needs. In many disease states, activity could be dysregulated.
Controlling IGDs presents a transformative opportunity to control genomic activation with precision therapeutics.
We have rigorously explored this avenue of therapeutic approach, with the goal of exerting regulatory genomic activation to manage or potentially cure disease.
THE IGD: KEY REGULATOR OF
“I WAS TRYING TO ANSWER A REAL FUNDAMENTAL QUESTION—HOW DO GENES AND THEIR REGULATORY ELEMENTS INTERACT?
WE MADE THE ARGUMENT THAT [IGDs] WERE THE FUNDAMENTAL UNIT OF GENOMIC CONTROL.”
—RICK YOUNG, PROFESSOR OF BIOLOGY, MIT & WHITEHEAD INSTITUTE OMEGA THERAPEUTICS BOARD OF DIRECTORS
IGDs are three-dimensional structures resulting from tertiary folds of the DNA. These structures appear as loops that encompass one or more genes and their associated regulatory elements. Just as a thermostat can regulate temperature, the IGDs act as a GenomStats™ of the human genome to regulate genomic activity.
In 2017, the anatomy and functional role of IGDs were discovered and documented by Young and Jaenisch, who observed their role in determining whether and to what degree genes are expressed. They demonstrated that perturbing the configuration of the IGDs could affect genomic activity. Omega Therapeutics has demonstrated that controlling the structure of IGDs and targeting regulatory elements within IGDs tune genomic activity.
The human genome is comprised of approximately 15,000 IGDs. Omega Therapeutics understands the location and physical configuration of each IGD, contributing a new level of scientific understanding about the human genome.
REGULATING THE GENOME
BY ADJUSTING THE GENOMSTATS™
Scientists at Omega Therapeutics have investigated the biological root of many diseases, determining which IGDs are implicated and what kind of intervention — upregulation or downregulation — is necessary for therapeutic effect.
Our proprietary Omega Controllers™ are engineered to precisely tune IGDs, the fundamental regulators of the genome, or GenomStats™, effectively directing the genome to treat disease.
While they are targeted with high specificity, Omega Controllers™ do not alter the nucleic acid sequences like many other methods of gene therapies that modify the genetic code.
Omega Controllers™ are modular, comprising of three elements: a functional domain (e.g., epigenetic in nature), which is engineered for optimal genomic tuning of the targeted GenomStats™ either up or down; a targeting domain (e.g., DNA/RNA binding), which binds to the specific genomic address considered; and an optimized delivery system, which delivers to the specific tissue or cell type.
Omega Controllers™ are a transformative therapeutics class that are designed to deliver precision genomic control to treat and potentially cure diseases by regulating the genome itself.
SPECIFIC, PRECISE, DURABLE