Novel Therapeutic Applications of Gene Editing Technology

Publication ID: 24-11857641_0002_PTD

Published: October 28, 2025

Category:New Applications & Use Cases

Legal Citation

pr1or.art Inc., “Novel Therapeutic Applications of Gene Editing Technology,” Published Technical Disclosure No. 24-11857641_0002_PTD, Published October 28, 2025, available at https://archive.pr1or.art/24-11857641_0002_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. 11,857,641.

Summary of the Inventive Concept

This inventive concept expands the application of gene editing technology to new therapeutic areas, including hearing loss, age-related macular degeneration, peripheral neuropathy, sickle cell disease, and muscular dystrophy, providing new hope for patients with these debilitating conditions.

Background and Problem Solved

The original patent focused on treating mucopolysaccharidosis type I (MPS I) using a combination of AAV vectors. However, the core technology has broader applicability, and this inventive concept addresses the limitations of the original patent by exploring new therapeutic areas where gene editing can make a significant impact.

Detailed Description of the Inventive Concept

The new claims describe systems and methods for treating various diseases using gene editing technology, including engineered zinc finger nucleases (ZFNs) and AAV vectors. For example, the composition of ZFNs can be used to site-specifically integrate a corrective copy of a hearing-related gene into inner ear cells to restore hearing. Similarly, AAV vectors encoding an enzyme that degrades lipofuscin can be administered to prevent and/or treat age-related macular degeneration. These new applications leverage the core technology to address unmet medical needs in diverse fields.

Novelty and Inventive Step

The new claims introduce novel and non-obvious applications of gene editing technology, expanding its therapeutic reach beyond MPS I. The inventive step lies in the recognition of the technology's broader potential and the development of new systems and methods tailored to specific diseases.

Alternative Embodiments and Variations

Alternative embodiments may include the use of CRISPR-Cas9 or other gene editing tools, different vector systems, or various routes of administration. Variations may involve targeting different genes or using combination therapies to enhance treatment outcomes.

Potential Commercial Applications and Market

This inventive concept has significant commercial potential, as it opens up new markets in hearing loss, ophthalmology, neurology, and hematology. The addressable market is substantial, with millions of patients worldwide suffering from these diseases, and the technology has the potential to transform treatment paradigms and improve patient outcomes.

CPC Classifications

Section	Class	Group
A	A61	A61K48/005
A	A61	A61K38/47
A	A61	A61P3/00
C	C12	C12Q1/34
C	C12	C12Y301/06013
C	C12	C12Y302/01076
G	G01	G01N33/66

Section 103 Obviousness Analysis (PHOSITA)

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Field of Art

Gene therapy and molecular medicine, specifically focusing on AAV vector-based gene editing technologies for treating genetic disorders, with expertise in molecular biology, virology, genetic engineering, and therapeutic interventions

Person of Ordinary Skill (PHOSITA) Profile

A molecular biologist or geneticist with advanced training, familiar with gene therapy techniques, AAV vector design, site-specific gene integration, and therapeutic applications across multiple genetic disorders

Obviousness Rationale

A PHOSITA would recognize the fundamental similarity between the source patent's AAV vector-based gene therapy for MPS I and the PTD's expanded applications, understanding that gene editing technologies can be systematically adapted across related genetic disorders by applying consistent molecular engineering principles and vector delivery strategies.

Obvious Combinations & Variations

Source Patent Element

AAV vector-based gene therapy targeting specific genetic disorders

PTD Variation

Applying AAV vector technology to hearing loss, macular degeneration, peripheral neuropathy, sickle cell disease, and muscular dystrophy

Obviousness Reasoning

Known technique of adapting gene therapy platforms to address different genetic conditions with predictable molecular engineering approaches

Source Patent Element

Site-specific gene integration using viral vectors

PTD Variation

Using zinc finger nucleases for targeted gene correction in hearing-related genes and hemoglobin beta gene

Obviousness Reasoning

Predictable extension of existing gene editing technologies to address specific genetic mutations across different disease contexts

Source Patent Element

Enzyme replacement strategy for reducing metabolic dysfunction

PTD Variation

Administering AAV vectors encoding enzymes to degrade lipofuscin or glycosaminoglycans in specific tissue contexts

Obviousness Reasoning

Routine application of enzyme replacement principles to novel therapeutic targets using established viral vector delivery mechanisms

Source Patent Element

Multi-vector gene therapy approach for complex genetic interventions

PTD Variation

Developing combination therapies targeting multiple genetic mechanisms in different disease states

Obviousness Reasoning

Obvious progression of gene therapy strategies from single to multi-vector approaches based on increasing understanding of genetic complexity

Source Patent Element

Therapeutic gene expression in specific cellular contexts

PTD Variation

Targeting gene expression in inner ear cells, retinal pigment epithelium, hematopoietic stem cells, and muscle tissue

Obviousness Reasoning

Predictable extension of gene therapy principles by applying consistent molecular engineering techniques across diverse tissue types

35 U.S.C. § 103 Summary: Based on the teachings of US Patent 11857641 and the published technical disclosure, a person having ordinary skill in the art would find the claimed variations in gene therapy applications obvious and predictable, as the fundamental molecular engineering principles and viral vector delivery strategies remain consistent across different genetic disorders, thereby rendering such extensions unpatentable under 35 U.S.C. Section 103.

Original Patent Information

Patent Number	US 11,857,641
Title	Method for the treatment of mucopolysaccharidosis type I
Assignee(s)	Sangamo Therapeutics, Inc.