CFB Inhibitors Modulate Complement Pathway to Reduce C3 Deposits

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Solution Overview

Problem

Dysregulation of the complement alternative pathway leads to severe diseases, and existing treatments are inadequate in addressing the hyperactivity and associated tissue damage, particularly in the eyes and kidneys.

Innovation Solution

Administration of a Complement Factor B (CFB) specific inhibitor to inhibit the expression and accumulation of C3 deposits, thereby modulating the complement alternative pathway activity.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If the complement alternative pathway is continuously activated at a low tickover level, then homeostasis is maintained, but dysregulation beyond this level causes severe injury and tissue damage

Engineering Contradiction:
ImprovehomeostasisVSAvoidtissue damage
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent applies parameter changes by targeting and modulating the activity level of the complement alternative pathway. By administering CFB specific inhibitors, the pathway activity is changed from a potentially harmful hyperactive state to a controlled state, reducing tissue damage while maintaining necessary homeostatic function.

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent uses CFB specific inhibitors as intermediary substances that mediate between the complement alternative pathway and the host tissue. These inhibitors act as intermediaries to control pathway activity, preventing direct harmful interactions while allowing beneficial homeostatic functions to continue.

Inventive Principle:
Principle #24Intermediary (Mediator)

2Measurement precision

If C3 deposits accumulate in the eye and kidney, then complement alternative pathway hyperactivity is indicated, but existing treatments are inadequate in addressing this accumulation

Engineering Contradiction:
ImproveC3 deposit detectionVSAvoidtreatment effectiveness
Core Design Contradiction:
Measurement precisionVSEase of manufacture

Solution Approach 1:

The patent applies the taking out principle by specifically targeting and removing C3 deposits from tissues through CFB specific inhibition. The treatment extracts and eliminates the harmful C3 deposits from the eye and kidney, addressing the root cause of complement pathway hyperactivity rather than merely treating symptoms.

Inventive Principle:
Principle #2Taking out (Extraction)

Solution Approach 2:

The patent changes the parameter of C3 deposit accumulation by administering CFB specific inhibitors that modify complement pathway activity. This parameter change reduces C3 deposition in target tissues, providing a measurable improvement in treatment effectiveness for complement-related diseases.

Inventive Principle:
Principle #35Parameter changes

Data Source

PatentUS20240247268A1Modulators of Complement Factor B
Publication Date: 2024.07.25 IONIS PHARMACEUTICALS INC
  • US20240247268A1 patent drawing
  • US20240247268A1 patent drawing
  • US20240247268A1 patent drawing

AI summary

The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a Complement Factor B (CFB) specific inhibitor to a subject.