CAR-NK Cell DNA Crosslinking to Limit Post-Infusion Proliferation

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Solution Overview

Problem

Existing CAR-NK cell therapies face safety concerns such as on-target/off-tumor effects, graft-versus-host-disease, cytokine release syndrome, and toxicity to normal tissues, necessitating improved methods and compositions for safer administration.

Innovation Solution

Introduce interstrand crosslinks and/or adducts into the genomic nucleic acids of CAR-NK cells through reaction with a nucleic acid targeting compound to inhibit further division while maintaining immunologic function, using compounds like psoralen activated by UVA irradiation.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Quantity of substance

If CAR-NK cells are expanded in vitro to increase therapeutic dosage, then the quantity of cells increases, but the risk of uncontrolled proliferation and off-target effects in the recipient increases

Engineering Contradiction:
Improvequantity of CAR-NK cellsVSAvoidoff-target effects and toxicity
Core Design Contradiction:
Quantity of substanceVSObject-affected harmful factors

Solution Approach 1:

The patent applies preliminary action by introducing nucleic acid targeting compounds (such as psoralen) and UV irradiation to the CAR-NK cells before administration to the recipient. This pre-treatment creates DNA crosslinks and adducts that permanently inhibit cell division, ensuring that the cells cannot proliferate uncontrollably after infusion. The harmful proliferation risk is addressed in advance, allowing safe expansion to therapeutic dosages without compromising safety.

Inventive Principle:
Principle #10Preliminary action

2Reliability

If nucleic acid targeting compounds are introduced to inhibit cell proliferation, then safety against uncontrolled division improves, but the ability of cells to divide and expand therapeutically is reduced

Engineering Contradiction:
Improvesafety of CAR-NK cell therapyVSAvoidcell proliferation capacity
Core Design Contradiction:
ReliabilityVSProductivity

Solution Approach 1:

The patent applies local quality by differentiating between two functional requirements: therapeutic expansion (which requires proliferation) and long-term safety (which requires proliferation inhibition). The nucleic acid targeting compounds are introduced at controlled levels and timings to achieve local quality control - allowing sufficient cell expansion to reach therapeutic dosages while simultaneously establishing permanent proliferation limits. This creates a spatial and temporal distinction between the expansion phase and the safety enforcement phase.

Inventive Principle:
Principle #3Local quality

Solution Approach 2:

The patent applies parameter changes by carefully controlling the concentration, timing, and type of nucleic acid targeting compounds (such as psoralen) to modulate the degree of DNA modification. By adjusting these parameters, the invention achieves an optimal balance where enough cells are generated for therapeutic effect while sufficient DNA crosslinks are formed to prevent future uncontrolled proliferation. The parameters of compound concentration, UV irradiation dose, and treatment timing are optimized to simultaneously satisfy both productivity and safety requirements.

Inventive Principle:
Principle #35Parameter changes

3Reliability

If CAR-NK cells are activated to enhance cytotoxic function, then efficacy against target cells improves, but the risk of cytokine release syndrome and GVHD increases

Engineering Contradiction:
Improveefficacy of CAR-NK cell therapyVSAvoidcytokine release syndrome and GVHD
Core Design Contradiction:
ReliabilityVSObject-generated harmful factors

Solution Approach 1:

The patent applies preliminary action by introducing nucleic acid targeting compounds (such as psoralen) and UV irradiation to the CAR-NK cells before administration to the recipient. This pre-treatment creates DNA crosslinks and adducts that permanently inhibit cell division, ensuring that the cells cannot proliferate uncontrollably after infusion. The harmful proliferation risk is addressed in advance, allowing safe expansion to therapeutic dosages without compromising safety.

Inventive Principle:
Principle #10Preliminary 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 modified CAR-NK cells effectively target and destroy cancer cells or viral-infected cells with reduced proliferation, minimizing off-target effects and toxicity, thereby enhancing safety and efficacy of the therapy.

Implementation Method 1

using compounds like psoralen activated by UVA irradiation

Methodology Applied
Scientific EffectPhotochemical reaction: Photopolymerisation

Data Source

PatentUS20250388642A1Compositions and methods for improved NK cell therapies
Publication Date: 2025.12.25 CERUS CORP

AI summary

The present disclosure relates to the preparation and use of CAR-NK cells which are modified by a nucleic acid targeting compound.