Precision Neuroprotection and Editing Platform for Neurodegenerative Diseases

Publication ID: 24-11857534_0010_PTD

Published: October 28, 2025

Category:Future Evolutions & Paradigm Shifts

Legal Citation

pr1or.art Inc., “Precision Neuroprotection and Editing Platform for Neurodegenerative Diseases,” Published Technical Disclosure No. 24-11857534_0010_PTD, Published October 28, 2025, available at https://archive.pr1or.art/24-11857534_0010_PTD

This technical disclosure describes improvements that would be readily apparent to a Person Having Ordinary Skill In The Art (PHOSITA) when considered in combination with the foundational architecture disclosed in U.S. Patent No. 11,857,534.

Summary of the Inventive Concept

A next-generation platform integrating machine learning, gene editing, and targeted nanoparticle delivery to revolutionize the diagnosis and treatment of neurodegenerative diseases, such as ALS and FTD.

Background and Problem Solved

The original patent disclosed the inhibition of dipeptide repeat proteins (DRPs) using Type I PRMT inhibitors, which has limitations in terms of precision diagnosis, treatment efficacy, and patient-specific variability. The new inventive concept addresses these limitations by envisioning a comprehensive platform that combines advanced diagnostic tools, precision gene editing, and targeted neuroprotective agents to provide personalized treatment strategies for patients with neurodegenerative diseases.

Detailed Description of the Inventive Concept

The platform consists of four interconnected modules: (1) a machine learning module trained on a dataset of DRP profiles to identify specific patterns correlated with disease progression and predict optimal treatment strategies; (2) a CRISPR-Cas9 system for in vivo editing of the C9ORF72 gene to correct the GGGGCC repeat expansion and prevent DRP toxicity; (3) a nanoparticle-formulated Type I PRMT inhibitor engineered to cross the blood-brain barrier and selectively target motor neurons; and (4) a personalized medicine platform that generates customized treatment plans based on patient-specific DRP profiles. The platform enables the creation of disease-modeling iPSC lines for drug discovery and development.

Novelty and Inventive Step

The new inventive concept introduces a paradigm shift in the treatment of neurodegenerative diseases by integrating machine learning, gene editing, and targeted nanoparticle delivery to provide personalized treatment strategies. The invention is novel and non-obvious in its combination of advanced diagnostic tools, precision gene editing, and targeted neuroprotective agents, which overcome the limitations of the original patent.

Alternative Embodiments and Variations

Alternative embodiments of the platform could include the use of different machine learning algorithms, gene editing tools, or nanoparticle formulations. Variations of the platform could also be developed for specific neurodegenerative diseases, such as Alzheimer's disease or Parkinson's disease.

Potential Commercial Applications and Market

The precision neuroprotection and editing platform has significant commercial potential in the pharmaceutical and biotechnology industries, with a projected market size of over $10 billion by 2025. The platform could be licensed to pharmaceutical companies, biotech startups, or research institutions, and could also be used to develop new business models for personalized medicine and precision healthcare.

Section 103 Obviousness Analysis (PHOSITA)

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Field of Art

Neurodegenerative disease research, specifically focusing on molecular mechanisms of ALS and FTD, involving gene editing, protein modification, and targeted therapeutic interventions

Person of Ordinary Skill (PHOSITA) Profile

A researcher with advanced degrees in molecular biology, neuroscience, or biomedical engineering, possessing expertise in CRISPR technologies, protein biochemistry, and computational biology techniques

Obviousness Rationale

A person of ordinary skill would recognize that the PTD represents a logical progression of the source patent's core teachings about dipeptide repeat protein (DRP) toxicity, extending the original inhibition strategy through predictable technological advancements in gene editing, machine learning, and targeted drug delivery. The PTD systematically addresses limitations in the original patent by introducing complementary approaches to DRP management. These variations represent incremental innovations that would be apparent to a skilled practitioner familiar with emerging neurodegenerative disease research methodologies.

Obvious Combinations & Variations

Source Patent Element

Type I PRMT inhibitor for decreasing DRP cellular toxicity

PTD Variation

Nanoparticle-formulated PRMT inhibitor engineered to cross blood-brain barrier

Obviousness Reasoning

Developing targeted drug delivery mechanisms is a known technique in pharmaceutical research, representing a predictable optimization of the original therapeutic approach

Source Patent Element

Identification of C9ORF72 gene GGGGCC repeat expansion

PTD Variation

CRISPR-Cas9 system for precise in vivo gene editing to correct repeat expansion

Obviousness Reasoning

CRISPR gene editing is a well-established technique for genetic modification, and applying it to correct known genetic mutations represents an obvious solution for a skilled researcher

Source Patent Element

Method of decreasing DRP cellular toxicity

PTD Variation

Machine learning module for predicting DRP patterns and treatment strategies

Obviousness Reasoning

Computational approaches for disease pattern recognition are standard in modern biomedical research, representing a predictable application of computational techniques to existing biological understanding

Source Patent Element

Treatment of neurological disorders involving DRPs

PTD Variation

Personalized medicine platform generating patient-specific treatment plans

Obviousness Reasoning

Precision medicine approaches are a known trend in therapeutic development, representing an obvious extension of existing disease management strategies

Source Patent Element

Identification of DRP toxicity mechanisms

PTD Variation

Generation of disease-modeling iPSC lines for drug discovery

Obviousness Reasoning

Creating cellular models for research is a standard technique in translational medicine, representing a predictable approach to further understanding disease mechanisms

35 U.S.C. § 103 Summary: Based on the teachings of US Patent 11857534 and the comprehensive technical disclosure herein, a person of ordinary skill in the art would find the claimed innovations obvious and lacking inventive step. The systematic integration of gene editing, machine learning, and targeted drug delivery represents a predictable combination of known techniques in neurodegenerative disease research, thereby rendering subsequent claims of novelty unpatentable under 35 U.S.C. § 103.

Original Patent Information

Patent Number	US 11,857,534
Title	Inhibition of dipeptide repeat proteins
Assignee(s)	ALS Therapy Development Institute