At GENFIT, as world-leading experts in gene regulation and nuclear receptors, we leverage the potential of these therapeutic targets to develop next generation treatments.
As transcription factors, nuclear receptors are intracellular receptors that participate in numerous fundamental physiological mechanisms. For example, they regulate key biological functions such as lipid and glucose metabolism, as well as the proliferation and differentiation of certain cell types.
Nuclear receptors as therapeutic targets
The nuclear receptors are important for homeostatic control and for adaptation to environmental variation. They thus participate in the molecular mechanisms whose dysregulation is associated with metabolic diseases (dyslipidemia, diabetes), inflammatory diseases, and certain types of cancer.
The therapeutic potential of certain nuclear receptors is already exploited by the pharma industry, such that they are the molecular target of approximately 13% of authorized medications (for example, estrogens, glucocorticoids, androgens, vitamin D, fibrates). The orphan nuclear receptors, for which no natural ligand has been identified, represent another potential source of therapeutic targets.
ROR: example of key nuclear receptors targeted at GENFIT
The nuclear receptors ROR (α, β, γ) are implicated in key physiological processes such as the regulation of lipid and glucose metabolism. They also participate in the regulation of certain inflammatory responses. The identification and characterization of synthetic ligands for these nuclear receptors thus represents a therapeutic opportunity for the treatment of certain metabolic and inflammatory diseases.
Based on the understanding of the physiopathological implications of ROR in diseases of interest for GENFIT, we are exploring a novel therapeutic approache related to RORγ, in the TGFTX1 program.