CPT-2008 Small-Molecule Compound for Mitochondrial ROS Control

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Solution Overview

Problem

Mitochondrial dysfunction is associated with a wide range of human diseases, including cancer, neurodegenerative diseases, and non-neurological disorders, with unclear mechanisms and a lack of effective treatments.

Innovation Solution

The use of CPT-2008, a 6-chloro-3-(2,4-dichloro-5-methoxyphenyl)-2-mercapto-7-methoxyquinazolin-4(3H)-one compound, or its derivatives, to alter mitochondrial reactive oxygen species (ROS) generation, electron transport chain activity, and mitochondrial morphology, thereby addressing mitochondrial dysfunction.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If conventional treatments are used for mitochondrial dysfunction, then some disease symptoms may be addressed, but the underlying mitochondrial dysfunction and ROS generation remain untreated

Engineering Contradiction:
Improveeffectiveness of treatmentVSAvoidROS generation
Core Design Contradiction:
ReliabilityVSObject-generated harmful factors

Solution Approach 1:

The patent applies this principle by using the harmful ROS generation as a target for treatment. CPT-2008 is designed to specifically inhibit mitochondrial ROS generation while maintaining electron transport chain function. The compound converts the harmful effect (excessive ROS) into a therapeutic target, allowing selective intervention in pathological ROS production without disrupting normal mitochondrial function.

Inventive Principle:
Principle #22Blessing in disguise (Convert harm into benefit)

Solution Approach 2:

CPT-2008 acts as an intermediary compound that mediates between the electron transport chain and ROS generation. The molecule structurally resembles ubiquinone (coenzyme Q10) and can interact with the electron transport chain complexes, particularly Complex I and III, to modulate electron flow and reduce leaky electron transfer that leads to ROS formation. This intermediary approach allows fine-tuned control of mitochondrial function.

Inventive Principle:
Principle #24Intermediary (Mediator)

2Reliability

If mitochondrial function is altered to treat disease, then disease symptoms improve, but off-target effects and toxicity may occur

Engineering Contradiction:
Improvedisease treatment efficacyVSAvoidoff-target effects
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent applies local quality by designing CPT-2008 to act specifically on mitochondrial ROS generation rather than affecting all cellular processes. The compound's structure and mechanism are tailored to target mitochondrial complexes I and III selectively, leaving other cellular pathways unaffected. This localized action reduces off-target effects while maintaining therapeutic efficacy for mitochondrial dysfunction-related diseases.

Inventive Principle:
Principle #3Local quality

Solution Approach 2:

CPT-2008 changes the parameter of ROS generation specifically in mitochondria without altering other critical parameters such as ATP production or cellular metabolism broadly. The compound modulates the electron transport chain to reduce superoxide formation while maintaining sufficient electron flow for ATP synthesis. This selective parameter change approach minimizes toxicity and off-target effects.

Inventive Principle:
Principle #35Parameter changes

Data Source

PatentUS12404251B2Small molecule compound
Publication Date: 2025.09.02 CEREPEUT INC
  • US12404251B2 patent drawing
  • US12404251B2 patent drawing
  • US12404251B2 patent drawing

AI summary

The present invention relates to compositions and methods for the treatment of cancer and other diseases associated with mitochondrial dysfunction, including but not limited to, neurodegenerative disease, brain injuries, and certain non-neurological disorders, using a novel compound, 6-chloro-3-(2,4-dichloro-5-methoxyphenyl)-2-mercapto-7-methoxyquinazolin-4(3H)-one, and derivatives thereof.