RGX-104 is primarily investigated for its potential therapeutic applications in the field of oncology. It functions as a small molecule activator of the liver X receptor (LXR), which plays a critical role in regulating cholesterol homeostasis and immune responses. By activating LXR, RGX-104 has shown promise in enhancing the body's anti-tumor immune response. This is achieved through the upregulation of apolipoprotein E (ApoE), which in turn promotes the recruitment and activation of immune cells, such as dendritic cells and cytotoxic T cells, within the tumor microenvironment.

Preclinical studies suggest that RGX-104 may be effective in treating various cancers, particularly those resistant to conventional therapies, such as non-small cell lung cancer, melanoma, and ovarian cancer. Its ability to modulate the immune system and target tumor-associated macrophages makes it a potential candidate for combination therapies with immune checkpoint inhibitors or other immunotherapeutic agents. Research is ongoing to further evaluate its efficacy, safety, and potential as a novel cancer treatment.