scAAV2-shmTOR-SD Gene Therapy for Diabetic Retinopathy

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Solution Overview

Problem

Current treatments for diabetic retinopathy, such as anti-VEGF therapies, require frequent intraocular injections, causing pain, eye damage, and high costs, and not all patients respond effectively, necessitating a long-term therapeutic solution for this degenerative retinal disease.

Innovation Solution

Development of a gene therapeutic agent, scAAV2-shmTOR-SD, using an AAV-based vector to deliver shRNA that inhibits the mTOR protein, providing a single administration option for treating diabetic retinopathy, thereby reducing vascular leakage, retinal vascular degeneration, and neuronal degeneration.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If anti-VEGF therapies are administered via repeated intraocular injections, then VEGF suppression is achieved, but patient compliance deteriorates due to frequent procedures and pain

Engineering Contradiction:

ImproveVEGF suppression efficacyVSAvoidpatient compliance

Core Design Contradiction:

ReliabilityVSEase of operation

Solution Approach 1:

The patent replaces repeated mechanical intraocular injections with a single injection of scAAV2-shmTOR-SD that provides long-term therapeutic effect through gene expression inhibition, eliminating the need for frequent procedures and improving patient compliance

Inventive Principle:

Principle #28Mechanics substitution (Replace mechanical system)

Solution Approach 2:

The scAAV2-shmTOR-SD vector provides continuous mTOR inhibition through sustained shRNA expression in retinal cells, maintaining therapeutic effect over time without requiring repeated administrations, thus resolving the contradiction between efficacy and compliance

Inventive Principle:

Principle #20Continuity of useful action

2Reliability

If protein-based anti-VEGF therapies are administered every 1-2 months, then VEGF is suppressed, but treatment cost and burden increase due to repeated administrations

Engineering Contradiction:

ImproveVEGF suppressionVSAvoidtreatment frequency

Core Design Contradiction:

ReliabilityVSLoss of time

Solution Approach 1:

The scAAV2-shmTOR-SD vector delivers mTOR inhibitory shRNA that begins acting immediately and provides long-term suppression, eliminating the need for repeated monthly injections and reducing treatment time loss

Inventive Principle:

Principle #10Preliminary action

Solution Approach 2:

The gene therapeutic agent establishes continuous mTOR inhibition through persistent vector expression in retinal cells, maintaining therapeutic effect indefinitely without requiring repeated administrations, thus resolving the contradiction between suppression reliability and treatment frequency

Inventive Principle:

Principle #20Continuity of useful action

3Reliability

If protein therapeutic agents are injected intravitreally, then therapeutic effect is achieved, but side effects increase due to repeated injections into the eyeball

Engineering Contradiction:

Improvetherapeutic efficacyVSAvoidside effects

Core Design Contradiction:

ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent substitutes repeated mechanical intravitreal injections with a single injection of scAAV2-shmTOR-SD that provides long-term effect through genetic inhibition, reducing the number of times the eyeball is punctured and thereby reducing side effects

Inventive Principle:

Principle #28Mechanics substitution (Replace mechanical system)

Solution Approach 2:

The gene vector provides continuous therapeutic effect through sustained mTOR inhibition without requiring repeated injections, thereby reducing cumulative side effects while maintaining therapeutic efficacy

Inventive Principle:

Principle #20Continuity of useful action

Applied Scientific Principles

This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.

Function Achieved in This Case

The scAAV2-shmTOR-SD agent effectively suppresses mTOR expression, offering a long-term therapeutic effect by inhibiting key symptoms of diabetic retinopathy, including macular edema and vascular leakage, with reduced side effects and treatment frequency compared to conventional methods.

Implementation Method 1

a recombinant vector including mTOR shRNA for mTOR inhibition

Methodology Applied

Scientific EffectRNA interference (shRNA):

Implementation Method 2

an AAV-based gene therapeutic agent showing a long-term effect through a single injection

Methodology Applied

Scientific EffectGene therapy:

Data Source

PatentUS20240218372A1COMPOSITION FOR TREATING DIABETIC RETINOPATHY CONTAINING scAAV2-shmTOR-SD (CRG-01)

Publication Date: 2024.07.04 CDMOGEN CO LTD

AI summary

The present disclosure relates to a composition for treating diabetic retinopathy, containing scAAV2-shmTOR-SD (CRG-01), a therapeutic method of suppressing expression of mTOR proteins to treat diabetic retinopathy, and a method of using an RNAi method by means of short hairpin RNA (shRNA) as a method of suppressing expression and activity of mTOR proteins, and development of a composition for treating diabetic retinopathy by performing delivery to retina using AAV2 and being expressed in the form of mTOR shRNA as a method for expressing mTOR shRNA in cells of the retina.