Functional Genetics’ core discovery technology, Random Homozygous Gene Perturbation (RHGP), provides a superior means to identify novel targets and pathways. RHGP can efficiently interrogate every gene in a cell, both for gain or loss of target function, and efficiently focus upon those targets that directly cause the desired experimental outcome.
RHGPcenters upon the use of a novel genetic element, known as a Gene Search Vector (GSV), which can probe the entire genome to up- or down-regulate each target in a cell. In doing so, the GSV can be efficiently monitored to allow FGI to identify causative events (ie target changes) that allow cells to display a desired phenotype. Through a strategy of ever-improving the technology through proprietary means, FGI is now capable of identifying relevant targets within days or weeks, rather than months.
The validation methodologies include the use of a reversible promoter in the GSV (which dramatically increases the efficiency of target identification) and the use of naïve systems (eg, siRNA or ectopic overexpression) to verify target relevance. These validation protocols are further strengthened by the use of targeted interventions to verify that intervention could translate into a therapeutic product.
Functional Genetics and our partners have successfully used RHGP to identify novel targets with applications to cancer (targets that cause chemotherapy resistance or metastasis), infectious disease (targets for broad-spectrum antivirals and antibiotics), Alzheimer’s disease (targets that cause amyloid plaques), manufacturing of biologics (pathways that increase the efficiency of monoclonal antibody production), and other indications.
Improvements in our technologies allow us to rapidly fill or refill our pipeline with innovative products. The Company has recently committed to expand the use of RHGP with partners to allow others to fully realize the potential of the RHGP technology for target discovery.