Personalized Neurodegenerative Disease Treatment Platform
Legal Citation
Summary of the Inventive Concept
A next-generation treatment platform for neurodegenerative diseases, integrating real-time monitoring, machine learning, and precision medicine to provide tailored treatment regimens and prevent disease onset.
Background and Problem Solved
The original patent's method of decreasing cellular toxicity caused by dipeptide repeat proteins (DRPs) has limitations in terms of personalized treatment and prevention of disease onset. The new inventive concept addresses these limitations by introducing a system that adapts to individual patients' needs and integrates multiple approaches to prevent and treat neurodegenerative diseases.
Detailed Description of the Inventive Concept
The new inventive concept comprises a personalized, adaptive dosing module that adjusts the administration of a Type I PRMT inhibitor based on real-time monitoring of DRP-induced toxicity in a patient's cells. Additionally, a machine learning algorithm integrates data from multiple sources to predict optimal treatment regimens. The system also includes a gene editing tool that selectively targets and corrects the C9ORF72 gene mutation, thereby eliminating the source of DRP production. Furthermore, a diagnostic assay detects DRP-induced toxicity in cells, and a genomics module identifies genetic variants associated with DRP production. The precision medicine algorithm recommends tailored treatment regimens based on an individual's genetic profile and DRP expression levels.
Novelty and Inventive Step
The new claims introduce a paradigm shift in treating neurodegenerative diseases by integrating real-time monitoring, machine learning, and precision medicine. The use of adaptive dosing, gene editing, and genomics-based diagnosis and treatment planning is novel and non-obvious compared to the original patent's method of decreasing cellular toxicity.
Alternative Embodiments and Variations
Alternative embodiments of the inventive concept could include the use of different types of inhibitors, gene editing tools, or diagnostic assays. Additionally, the platform could be adapted for treating other diseases characterized by protein misfolding or aggregation.
Potential Commercial Applications and Market
The personalized neurodegenerative disease treatment platform has significant commercial potential in the pharmaceutical and biotechnology industries, with potential applications in treating ALS, FTD, and other neurodegenerative diseases. The market for such a platform is substantial, with an estimated value of billions of dollars.
Original Patent Information
| Patent Number | US 11,857,534 |
|---|---|
| Title | Inhibition of dipeptide repeat proteins |
| Assignee(s) | ALS Therapy Development Institute |