Inhibition of Clathrin

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

This patent discloses a new class of compounds that inhibit the activity of clathrin, a key protein involved in clathrin-mediated endocytosis and cell division (mitosis). Specifically, the invention identifies new binding sites on the clathrin terminal domain and provides chemical inhibitors (such as pitstop 1 and pitstop 2) that selectively block clathrin function. These inhibitors can prevent clathrin-mediated uptake into cells and suppress cell division, making them useful for both research and therapeutic applications, including as potential treatments for diseases that involve aberrant cell growth, viral infections, neurological disorders, and more.

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

• As anti-cancer agents to inhibit cancer cell proliferation by blocking cell division (mitosis).
• As antiviral agents to prevent viral entry into cells by inhibiting clathrin-mediated endocytosis (e.g., for HIV, hepatitis C, Ebola, etc.).
• For the treatment of neurological conditions such as epilepsy, Alzheimer's disease, Parkinson's disease, and other neurodegenerative disorders, by modulating synaptic activity or endocytic pathways.
• As research tools to study the function of clathrin and the mechanisms of endocytosis and mitosis in cells.
• For treatment of cell proliferative disorders beyond cancer, such as psoriasis, scleroderma, and atherosclerosis.
• Potential therapy for neuropathic pain and seizure control by interfering with synaptic vesicle recycling in neurons.
• To block the cellular entry of pathogenic bacteria and toxins that utilize clathrin-mediated pathways.

BenefitsContent extracted from patent full text and abstract with AI.

• Provides the first specific small-molecule inhibitors that directly target clathrin function, offering selectivity compared to previous endocytosis inhibitors.
• Enables the rational design of further inhibitors due to detailed characterization of the clathrin binding site.
• Inhibitors can rapidly and reversibly block clathrin-mediated processes in cells, providing temporal control.
• Broad therapeutic potential across cancer, viral infections, neurological diseases, and more.
• Facilitates novel approach to anti-cancer treatment by targeting cell division at a fundamental cellular level.
• Potential for fewer side-effects in neurological applications due to activity-dependent inhibition (e.g., targeting overactive neurons during seizures).
• Can serve as powerful research tools for dissecting cell biology, opening new experimental avenues.

Technical Classifications (CPCs)

Inventors & Applicants

Patent Abstract

Inhibitors are provided for inhibiting the activity of clathrin. In particular, binding site(s) on the clathrin terminal doman (TD) for binding inhibitors have been identified. The binding sites are defined by amino acid(s) in the group Ile 52, Ile 62, Ile 80, Phe 91, and Ile 93 of the clathrin TD (SEQ ID No. 1). In at least some forms, the binding site may be further defined by amino acid(s) in the group Ile 66, Arg 64, Leu 82 andf Lys 96 of SEQ ID No. 1, or by Val 50 of SEQ ID No.1. There are also provided methods for prophylaxis or treatment of disease and conditions responsive to the inhibition of clathrin.