AAV Capsid Ratio Assay With Regeneration to Eliminate Ghost Peaks

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Solution Overview

Problem

Existing methods for determining the relative abundance of intact adeno-associated virus (AAV) capsid components in recombinant AAV particles are prone to analytical errors due to ghost peaks, which affect product quality and consistency, especially in good manufacturing practice (GMP) assays.

Innovation Solution

A method involving system regeneration, equilibration, and sample separation processes using anion exchange chromatography with specific mobile phases and wash solutions to accurately separate and quantify intact empty and full AAV capsids, minimizing carry-over and ghost peaks.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Measurement precision

If conventional anion exchange chromatography is used for AAV capsid separation, then separation of empty and full capsids is achieved, but ghost peaks appear causing analytical errors

Engineering Contradiction:
Improvequantification accuracyVSAvoidresult consistency
Core Design Contradiction:
Measurement precisionVSReliability

Solution Approach 1:

The method performs preliminary system regeneration and equilibration steps before actual sample analysis. The AEX column is regenerated with high salt concentration mobile phase and equilibrated with low salt concentration mobile phase to ensure consistent baseline conditions, preventing ghost peaks from carrying over between runs

Inventive Principle:
Principle #10Preliminary action

Solution Approach 2:

The method systematically optimizes and controls multiple parameters including mobile phase pH (7.5-9.0), salt concentration gradients (NaCl or TMAC), buffer composition (BTP), and temperature to achieve consistent separation of empty and full capsids while minimizing analytical artifacts

Inventive Principle:
Principle #35Parameter changes

2Illumination intensity

If higher concentrations of MgCl2 are used in mobile phase, then peak intensity increases, but structural integrity of AAV capsids may be compromised

Engineering Contradiction:
Improvepeak intensityVSAvoidcapsid structural integrity
Core Design Contradiction:
Illumination intensityVSStability of the object's composition

Solution Approach 1:

The method optimizes MgCl2 concentration within a specific range (1-3 mM) to achieve sufficient peak intensity while maintaining capsid structural integrity. This parameter optimization balances detection sensitivity with preservation of viral particle integrity throughout the chromatographic process

Inventive Principle:
Principle #35Parameter changes

3Measurement precision

If NaCl is used as mobile phase salt, then separation is achieved, but carry-over between samples occurs

Engineering Contradiction:
Improveseparation resolutionVSAvoidsample carry-over
Core Design Contradiction:
Measurement precisionVSLoss of substance

Solution Approach 1:

The method implements a dual-mode mobile phase system where high salt concentration (1.5-2.5 M NaCl or 0.5-1.0 M TMAC) is used for effective capsid separation, followed by systematic column regeneration and equilibration steps that restore the column to baseline conditions, preventing carry-over to subsequent samples

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The method maintains continuous column regeneration and equilibration processes between sample injections, ensuring the column remains in a consistent, clean state ready for the next analysis without allowing sample residues to persist

Inventive Principle:
Principle #20Continuity of useful 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 method provides sensitive and quantitative characterization of AAV capsid components, ensuring product quality and consistency by reducing carry-over and ghost peaks, thereby enhancing the accuracy and reproducibility of AAV capsid quantification.

Implementation Method 1

separating the intact empty AAV capsids from the intact full AAV capsids by anion exchange chromatography

Methodology Applied
Scientific EffectAnion exchange chromatography: Ion Exchange

Implementation Method 2

washing liquid carrying components of the LC instrument with the wash solution

Methodology Applied
Scientific EffectChromatographic washing: Chromatography

Data Source

PatentUS12571797B2Liquid chromatography assay for determining AAV capsid ratio
Publication Date: 2026.03.10 REGENERON PHARMACEUTICALS INC

AI summary

Methods for determining the relative abundance of intact adeno-associated virus (AAV) capsid components in a sample of recombinant AAV particles are disclosed. In embodiments, the methods include a system regeneration process that minimizes or eliminates the presence of ghost peaks to maximize analytical accuracy and ensure product quality and consistency.