LC-MS RNA Capping Analysis Without Radiolabels

Resolve Bottlenecks,
Find Innovative Solutions
Generate Solutions

Solution Overview

Problem

Current methods for characterizing the 5' cap of mRNA are limited by the need for radiolabels and are not suitable for high-throughput analysis, particularly in the context of therapeutic mRNA development, where rapid and accurate characterization of in vitro transcribed mRNA is required.

Innovation Solution

A radiolabel-free method using nonradiolabeled tagged probes and liquid chromatography coupled with mass spectrometry (LC-MS) for identifying and quantifying the 5' end cap of mRNA, allowing for the determination of capping efficiency and orientation without the need for radiolabels, utilizing enzymes like RNAse H and 5' RNA pyrophosphohydrolase (RppH) for cleavage and analysis.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Measurement precision

If radiolabeled detection methods are used for 5' cap characterization, then measurement sensitivity is improved, but safety hazards and regulatory complexity increase due to radiolabel requirements

Engineering Contradiction:
Improvedetection sensitivityVSAvoidradiation safety hazards
Core Design Contradiction:
Measurement precisionVSObject-affected harmful factors

Solution Approach 1:

The patent replaces expensive and hazardous radiolabeled compounds with inexpensive, non-radioactive alternative labels such as fluorescent dyes, biotin, or mass-tagged moieties. These alternative labels provide sufficient detection sensitivity without the safety hazards of radiation, allowing for disposable, safer assay systems that can be easily handled and disposed of without special radiological precautions

Inventive Principle:
Principle #27Cheap short-living objects (Disposable)

Solution Approach 2:

The patent substitutes the radioactive detection mechanism with non-radioactive detection systems such as fluorescence detection, chemiluminescence, or mass spectrometry. This replacement eliminates the harmful radiation while maintaining or improving measurement precision through highly sensitive optical or mass-based detection methods

Inventive Principle:
Principle #28Mechanics substitution (Replace mechanical system)

2Measurement precision

If enzymatic digestion followed by PAGE or HPLC with radiolabel detection is used, then 5' cap characterization accuracy is improved, but processing throughput is reduced

Engineering Contradiction:
Improvecharacterization accuracyVSAvoidsample processing throughput
Core Design Contradiction:
Measurement precisionVSProductivity

Solution Approach 1:

The patent segments the complex analytical process into modular steps: (1) enzymatic cleavage to release 5' caps, (2) affinity capture of cleaved products using solid support, and (3) parallel detection of multiple samples. This segmentation allows for streamlined processing and potential automation, improving throughput while maintaining characterization accuracy

Inventive Principle:
Principle #1Segmentation

Solution Approach 2:

The patent introduces solid support affinity capture as an intermediary step between enzymatic digestion and detection. This intermediary step concentrates the cleaved 5' cap products from multiple samples onto solid phases, enabling efficient washing and detection while facilitating high-throughput processing by allowing parallel handling of multiple samples

Inventive Principle:
Principle #24Intermediary (Mediator)

3Measurement precision

If sequence specific RNAse H probes are used to cleave 5' end sections, then capping efficiency measurement is improved, but the method remains limited to radiolabeled detection

Engineering Contradiction:
Improvecapping efficiency measurementVSAvoidradiolabel dependency
Core Design Contradiction:
Measurement precisionVSObject-affected harmful factors

Solution Approach 1:

The patent changes the detection parameter from radioactive signal to non-radioactive signals such as fluorescence intensity, chemiluminescence signal, or mass-to-charge ratio. This parameter change maintains the precision of capping efficiency measurement while eliminating the harmful radiolabel dependency, allowing the same RNAse H probe cleavage methodology to be used with safe alternative detection modes

Inventive Principle:
Principle #35Parameter changes

4Measurement precision

If chromatographic resolution and retention time of standards are used for identification, then cap structure identification is achieved, but the method lacks sensitivity and throughput for therapeutic mRNA characterization

Engineering Contradiction:
Improvecap structure identificationVSAvoidanalysis throughput
Core Design Contradiction:
Measurement precisionVSProductivity

Solution Approach 1:

The patent uses solid support affinity capture as an intermediary to concentrate and pre-purify 5' cap structures from complex mRNA samples before analysis. This intermediary step enhances the sensitivity of subsequent detection methods and enables higher throughput by reducing sample complexity and allowing for automated processing and detection of multiple samples

Inventive Principle:
Principle #24Intermediary (Mediator)

Solution Approach 2:

The patent substitutes traditional chromatographic detection with more sensitive and higher-throughput detection methods such as mass spectrometry, fluorescence detection, or chemiluminescence. These replacement detection systems provide both the sensitivity needed for therapeutic mRNA characterization and the throughput capacity for rapid analysis of multiple samples

Inventive Principle:
Principle #28Mechanics substitution (Replace mechanical system)

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

Enables accurate and high-resolution identification of 5' cap structures and capping efficiencies in mRNA samples, facilitating the analysis of therapeutic mRNA preparations with improved throughput and sensitivity, capable of detecting uncapped species and determining capping efficiencies across various mRNA lengths and modifications.

Implementation Method 1

treating the duplex polynucleotide with RNAse H, thus cleaving the 5' end of the target RNA

Methodology Applied
Scientific EffectEnzymatic cleavage: Enzyme

Implementation Method 2

isolating the duplex polynucleotide, using a surface coated substrate that is coated with a reagent that binds to the nonradiolabeled tagged probe

Methodology Applied
Scientific EffectAdsorption: Adsorption

Implementation Method 3

analyzing the single-stranded fragment of the 5' end of the target RNA by liquid chromatography/mass spectrometry (LC-MS)

Methodology Applied
Scientific EffectChromatography: Chromatography

Implementation Method 4

analyzing the single-stranded fragment of the 5' end of the target RNA by liquid chromatography/mass spectrometry (LC-MS); identifying the 5' end cap

Methodology Applied
Scientific EffectMass spectrometry:

Data Source

PatentEP3387150B1Label-free analysis of RNA capping efficiency using rnase h, probes and liquid chromatography/mass spectrometry
Publication Date: 2019.10.02 NOVARTIS AG
  • EP3387150B1 patent drawingFigure 1
  • EP3387150B1 patent drawingFigure 2
  • EP3387150B1 patent drawingFigure 3

AI summary

Methods to qualitatively and quantitatively determine mRNA capping, and to determine 5' capping efficiency and 5' cap identity in RNA samples, all without the need for radiolabels, by using tagged probes that are complementary to the 5' end of target RNA and RNAse H to cleave the 5' end of RNA, then using LC-MS to determine the 5' RNA products.