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Ref Type
Authors M Mita, A Mita, B Chmielowski, M Postow, E Hamilton, S Pant, Roger Waltzman, Eric Rowinsky, Michael Szarek, Foster Gonsalves, Isabel Kurth, Celia Andreu, Ilana Pollack, Daniel Mucida, Raissa Tanqueco, Sohail Tavazoie and Masoud Tavazoie
Title A phase 1 trial of RGX-104, a first-in-class immunotherapy targeting the liver-X nuclear hormone receptor (LXR), in patients with refractory malignancies
Journal Mol Cancer Ther
Abstract Text Mol Cancer Ther 2018;17(1 Suppl):Abstract nr B001


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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
RGX-104 RGX-104 1 1
Drug Name Trade Name Synonyms Drug Classes Drug Description
RGX-104 RGX-104 activates liver-X nuclear hormone receptor (LXR), resulting in ApoE expression and enhancement of antitumor immunity (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr B001).
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown Advanced Solid Tumor not applicable RGX-104 Phase I Actionable In a Phase I trial, RGX-104 demonstrated safety and preliminary efficacy, resulted in targeted gene expression in 100% (6/6), and activation of anti-tumor immune response in 83% (5/6) of patients with solid tumors refractory to anti-PD-1 therapy, with one renal cancer and one melanoma patient achieved stable disease (Mol Cancer Ther 2018;17(1 Suppl):Abstract nr B001). detail...