Small Molecule Inhibitors of Guanine Nucleotide Exchange Factors of the Cytohesin Family

Simple SummaryContent extracted from patent full text and abstract with AI.

This patent describes low-molecular-weight compounds that selectively inhibit guanine nucleotide exchange factors (GEFs) of the cytohesin family. These compounds can be formulated as drugs for use in the treatment of autoimmune diseases, cancer (tumoral diseases), and for immunosuppression, for example in organ transplantation. The key innovation is the discovery of small molecule inhibitors that are cell-permeable, non-toxic at effective doses, and highly specific for cytohesin GEFs, which regulate essential cellular processes via small G-proteins (GTPases) and are implicated in several immune and cancer-related conditions.

Use CasesContent extracted from patent full text and abstract with AI.

• Treatment of autoimmune diseases such as rheumatoid arthritis, multiple sclerosis, type I and type II diabetes, psoriasis, Crohn's disease, and allergies.
• Treatment of cancers including lung, colon, prostate, lymphoma, bladder, breast, and uterine cancers.
• Immunosuppression in organ transplantation to prevent organ rejection.
• Targeted therapy to inhibit leukocyte adhesion and migration in various inflammatory conditions.
• Experimental use in biological research to study GEF/cytohesin function and signaling pathways.
• Development of diagnostic assays or screening platforms for new cytohesin inhibitors.

BenefitsContent extracted from patent full text and abstract with AI.

• First small-molecule, cell-permeable inhibitors specific to the cytohesin family of GEFs, filling a gap left by toxic or inefficient prior alternatives (e.g., Brefeldin A, RNA aptamers).
• Low toxicity at effective concentrations, broadening their therapeutic applicability and safety.
• High specificity to cytohesins, reducing side effects and off-target actions compared to less selective compounds.
• Effective at modulating immune cell activities, such as adhesion and migration, enabling control over immune and inflammatory responses.
• Versatile administration routes (oral, dermal, intravenous) due to excellent cell membrane permeability.
• Potential to treat a wide variety of immune-mediated and oncological diseases, as well as aid in transplantation medicine.

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Patent Abstract

The invention relates to drugs and the use thereof, wherein said drugs contain compounds selected from a group comprising general formulas (1), (2), (3) and (4) and/or enantiomers, diastereomers or the pharmaceutically acceptable salts thereof. Said drugs are used for treating autoimmune and tumoral diseases and/or for immunosuppresion.