CAR T-cell Persistence via CD28-4-1BB Costimulation

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Solution Overview

Problem

Current approaches for treating B-cell malignancies using genetically modified T cells with chimeric antigen receptors (CARs) face limitations such as limited in vivo expansion and rapid disappearance of CAR+ T cells, leading to disappointing clinical activity.

Innovation Solution

Development of a CAR comprising an antigen binding domain, a transmembrane domain, a costimulatory signaling region, and a CD3 zeta signaling domain, specifically designed to enhance the persistence and expansion of genetically engineered T cells, allowing them to target and eliminate tumor cells effectively.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If genetically modified T cells with CARs are used to treat B-cell malignancies, then tumor targeting capability is improved, but in vivo persistence and expansion of CAR+ T cells deteriorates

Engineering Contradiction:
Improvetumor targeting capabilityVSAvoidin vivo persistence of CAR+ T cells
Core Design Contradiction:
ReliabilityVSDuration of action of stationary object

Solution Approach 1:

The CAR structure is segmented into distinct functional domains: an extracellular antigen-binding domain (single-chain variable fragment), a transmembrane domain, and an intracellular signaling domain containing costimulatory elements (CD28 and/or 4-1BB) combined with CD3 zeta signaling motifs. This segmentation allows optimization of each domain's function to improve both tumor targeting and T cell persistence

Inventive Principle:
Principle #1Segmentation

Solution Approach 2:

The CAR is a composite molecular construct combining elements from different immune receptors: the antigen-binding specificity of antibodies, the transmembrane anchoring of T cell receptors, and costimulatory signaling domains from CD28, 4-1BB, and CD3 zeta. This composite structure integrates multiple signaling pathways to enhance T cell activation, survival, and persistence while maintaining tumor targeting

Inventive Principle:
Principle #40Composite materials

2Measurement precision

If conventional CAR designs are used, then tumor antigen recognition is achieved, but clinical activity and therapeutic effectiveness deteriorate

Engineering Contradiction:
Improvetumor antigen recognitionVSAvoidclinical activity
Core Design Contradiction:
Measurement precisionVSProductivity

Solution Approach 1:

The invention modifies critical parameters of the CAR structure, specifically incorporating dual costimulatory signaling domains (CD28 and 4-1BB) combined with CD3 zeta ITAM motifs in a specific configuration. These parameter changes in the signaling domain composition and architecture transform the CAR from a simple antigen recognizer into a potent activator of sustained T cell responses, dramatically improving clinical activity while maintaining antigen recognition precision

Inventive Principle:
Principle #35Parameter changes

Data Source

PatentEP3660029A1Use of chimeric antigen receptor-modified t cells to treat cancer
Publication Date: 2020.06.03 THE TRUSTEES OF THE UNIV OF PENNSYLVANIA
  • EP3660029A1 patent drawingFigure 1A
  • EP3660029A1 patent drawingFigure 1B
  • EP3660029A1 patent drawingFigure 1C

AI summary

The present invention provides compositions and methods for treating cancer in a human. The invention includes relates to administering a genetically modified T cell to express a CAR wherein the CAR comprises an antigen binding domain, a transmembrane domain, a costimulatory signaling region, and a CD3 zeta signaling domain.