Developing highly specific, patient-friendly treatments for a broad range of diseases
GalOmic™ encompasses our proprietary siRNA chemistry knowledge and enables generation of our potent and long-acting RNAi medicines.
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 GalOmic™ siRNA therapeutics take advantage of this process to silence disease-causing genes with precision. A GalOmic™ therapy 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.
With strong safety data and several FDA-approved treatments, siRNA therapies have demonstrated a higher probability of clinical success compared to other treatments. This means lower development risks and faster delivery of innovative, effective treatments to patients.
Targeting the liver
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.
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