GalOmicTM is our proprietary RNAi chemistry platform. It enables the generation of GalNAc-conjugated short interfering RNA (siRNA) drug candidates for hepatocyte-specific gene silencing via RNA interference (RNAi) of any target gene identified by HepNetTM. In combination with HepNet™, it offers end-to-end capabilities for rapid identification of novel disease-associated genes and drug candidate development of therapies with disease-modifying potential.
The GalOmicTM platform generates siRNA therapeutics by creating synthetic molecules to silence the expression of specific disease-associated genes, specifically in hepatocytes.
Our GalOmicTM medicines have several compelling benefits:
- Specific: Sparing other cell types in the body, helping to ensure that therapeutic levels of a drug are delivered to one cell type and target one gene
- Translatable: The test agent used for target validation becomes the clinical candidate
- Druggable: Can selectively silence any gene in hepatocytes
- Reproducible: New drug candidates can be generated quickly by changing the existing genetic sequence to target a different gene to treat a different disease
- Safe: Clinical studies have demonstrated siRNAs are safe and well tolerated
- Patient friendly: Infrequent subcutaneous administration and durable therapeutic effect. Typically, months before the next injection is needed.
What is RNAi?
RNAi is a biological process that occurs naturally within our cells to help regulate gene expression. The mechanism by which RNAi mediates its biological function is specific targeting of messenger RNA (mRNA) molecules, which carry the instructions cells need to make proteins out of the genetic manual encoded in our DNA.
Our siRNA therapeutics are synthetic molecules that harness this cellular process to silence the expression of disease-associated genes in a highly specific manner. An siRNA medicine designed to target a specific gene will, as part of an RNA-induced silencing complex (RISC), bind to its target mRNA and trigger its degradation. As a result, no disease-associated protein will be produced in the cell.
Hepatocyte-specific delivery
GalOmicTM achieves an additional level of specificity by coupling siRNA molecules to delivery systems for cell type-specific targeting. Our siRNA constructs are conjugated to GalNAc (N-Acetylgalactosamine) moieties, which mediate highly specific delivery to hepatocytes in the liver. As the liver is a highly active organ, hepatocyte targeting unlocks opportunities in a wide variety of therapeutic areas.
This cell type specificity spares other cell types in the body and ensures that therapeutic levels of the therapeutic reach target cells. Our siRNA medicines also incorporate optimised chemistries to maximise their therapeutic activity and, together with GalNAc-conjugation, enable subcutaneous administration.
Hepatocyte targeting unlocks many therapeutic possibilities
The liver plays a critical role in human health and is associated with a broad range of diseases with high unmet medical need. In addition, hepatocytes are highly influential both within, and beyond, the liver.
Over 12,000 genes are expressed in hepatocytes, meaning there is no shortage of targets amenable to our GalOmicTM RNAi platform. With the novel targets identified by our network-aware HepNetTM computational platform, we have the capability to treat a broad range of diseases with hepatocyte-specific delivery.
Related pages
Please visit the following pages for related information.
