Activatable Anti-CTLA-4 Antibodies for Tumor-Selective Binding

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Solution Overview

Problem

Current anti-CTLA-4 antibodies, such as ipilimumab, suffer from dose-limiting toxicities like colitis, necessitating the development of modified forms with reduced toxicity but comparable anti-tumor efficacy.

Innovation Solution

Development of activatable anti-CTLA-4 antibodies with a masking moiety and a cleavable moiety that are specifically designed to bind CTLA-4 only in the tumor microenvironment, reducing off-target binding and side effects while maintaining therapeutic efficacy.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If anti-CTLA-4 antibodies (e.g., ipilimumab) are used to treat tumors, then anti-tumor efficacy is improved, but dose-limiting toxicities (e.g., colitis) occur

Engineering Contradiction:
Improveanti-tumor efficacyVSAvoiddose-limiting toxicities
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent applies local quality by creating an activatable antibody that has different binding properties in different locations. The antibody is designed to be activated (remove masking moiety) specifically in the tumor microenvironment through proteolytic cleavage, allowing it to bind CTLA-4 and exert anti-tumor effects only at the tumor site, while remaining inactive in normal tissues to avoid toxicities like colitis.

Inventive Principle:
Principle #3Local quality

Solution Approach 2:

The patent implements preliminary action by incorporating a masking moiety that is pre-attached to the antibody's variable region. This masking moiety prevents off-target binding before the antibody reaches the tumor. Upon arrival at the tumor, the masking moiety is cleaved off by tumor-specific proteases, activating the antibody's CTLA-4 binding capability. This preliminary masking ensures safety during circulation and delivery.

Inventive Principle:
Principle #10Preliminary action

2Productivity

If anti-CTLA-4 antibodies bind CTLA-4 systemically, then T cell activation is promoted, but autoimmunity and inflammation increase

Engineering Contradiction:
ImproveT cell activationVSAvoidautoimmunity and inflammation
Core Design Contradiction:
ProductivityVSObject-generated harmful factors

Solution Approach 1:

The patent applies dynamics by creating an antibody that transitions from an inactive state (masked) to an active state (unmasked) based on the environmental conditions. The masking moiety is dynamically removed by proteolytic cleavage in the tumor microenvironment, allowing the antibody to activate T cells only where needed. This dynamic activation prevents systemic autoimmunity while maintaining tumor-specific T cell activation.

Inventive Principle:
Principle #15Dynamics

3Reliability

If anti-CTLA-4 antibodies are administered at high doses to maximize anti-tumor effect, then tumor regression improves, but toxicity increases

Engineering Contradiction:
Improvetumor regressionVSAvoidtoxicity
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent uses a masking moiety as an intermediary element that controls the antibody's activity. The masking moiety acts as a protective layer that prevents the antibody from binding CTLA-4 in normal tissues. Only in the tumor microenvironment, where proteases are present, is the masking moiety removed, allowing the antibody to exert its anti-tumor effects. This intermediary mechanism enables high anti-tumor efficacy without proportional increases in toxicity.

Inventive Principle:
Principle #24Intermediary (Mediator)

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 activatable antibodies retain anti-tumor activity by selectively binding CTLA-4 in tumors, minimizing side effects and enhancing treatment safety.

Implementation Method 1

a masking moiety (MM)... wherein the MM is selected from the group consisting of YV04, YV23, YV24, and YV39

Methodology Applied
Scientific EffectPhysical blocking:

Implementation Method 2

the masking moiety is removed by proteolytic cleavage of the cleavable moiety by tumor-specific proteases

Methodology Applied
Scientific EffectProteolytic cleavage: Decomposition (biological)

Implementation Method 3

Such activatable anti-human CTLA-4 antibodies have CTLA-4 binding activity in the tumor microenvironment

Methodology Applied
Scientific EffectAntibody-antigen binding:

Data Source

PatentEP3535300B1Activatable Anti-CTLA-4 antibodies and uses thereof
Publication Date: 2025.10.22 BRISTOL MYERS SQUIBB CO
  • EP3535300B1 patent drawingFigure 1A~1C
  • EP3535300B1 patent drawingFigure 2A~2C
  • EP3535300B1 patent drawingFigure 3A~3E

AI summary

Provided herein are activatable anti-human CTLA-4 antibodies comprising a heavy chain comprising a VH domain and a light chain comprising a masking moiety (MM), a cleavable moiety (CM), and a VL domain. Such activatable anti-human CTLA-4 antibodies have CTLA-4 binding activity in the tumor microenvironment, where the masking moiety is removed by proteolytic cleavage of the cleavable moiety by tumor-specific proteases, but exhibit greatly reduced binding to CTLA-4 outside the tumor. In this way, the activatable anti-human CTLA-4 antibodies of the present invention retain anti-tumor activity while reducing the side effects associated with anti-CTLA-4 activity outside the tumor.