Botulinum Toxin Chain Assembly Using GP41.1 Intein Splicing

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Solution Overview

Problem

Current methods for producing botulinum neurotoxin face challenges such as low yield, high production costs, and safety hazards due to the need for cleaving and linking of the heavy and light chains, which are difficult to express in soluble form in E. coli, and require additional processes for disulfide bond formation.

Innovation Solution

A method involving the expression and purification of botulinum toxin fragments using GP41.1 intein and a soluble partner, followed by protein trans-splicing to link the heavy and light chains without a soluble partner, and forming a disulfide bond between them, thereby increasing solubility and yield.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Productivity

If botulinum toxin is produced using traditional methods requiring cleavage and disulfide bond formation, then the toxin can be obtained, but the production yield decreases and costs increase

Engineering Contradiction:
Improveproduction yieldVSAvoidproduction complexity
Core Design Contradiction:
ProductivityVSEase of manufacture

Solution Approach 1:

The botulinum toxin is divided into separate heavy chain and light chain components that are expressed independently as fusion proteins with intein tags. These separate components are then purified and reassembled through protein trans-splicing, avoiding the need for proteolytic cleavage and subsequent disulfide bond formation steps required in traditional methods.

Inventive Principle:
Principle #1Segmentation

Solution Approach 2:

GP41.1 intein serves as an intermediary element that facilitates the assembly of heavy and light chains. The intein tags on separate toxin chains enable their specific pairing and trans-splicing to form the complete active toxin, replacing the need for endogenous proteases and oxidizing systems.

Inventive Principle:
Principle #24Intermediary (Mediator)

2Productivity

If botulinum toxin is expressed in E. coli, then production can be achieved, but the toxin cannot be expressed in soluble form

Engineering Contradiction:
Improveproduction capabilityVSAvoidsolubility
Core Design Contradiction:
ProductivityVSReliability

Solution Approach 1:

Soluble partner proteins are used as intermediaries to express the botulinum toxin chains in E. coli. The toxin chains are fused to soluble partner tags that enhance their solubility during expression, allowing the toxic components to be produced in a safe, soluble form that can later be purified and reassembled.

Inventive Principle:
Principle #24Intermediary (Mediator)

Solution Approach 2:

The toxin is segmented into separate chains that are individually expressed with soluble tags. This segmentation allows each component to be optimized for solubility and safety during expression, while the final active toxin is reconstituted through controlled trans-splicing of the intein tags.

Inventive Principle:
Principle #1Segmentation

3Manufacturing precision

If additional processes are added for disulfide bond formation, then correct toxin structure can be achieved, but the production process becomes more complex

Engineering Contradiction:
Improvestructural accuracyVSAvoidprocess complexity
Core Design Contradiction:
Manufacturing precisionVSDevice complexity

Solution Approach 1:

The GP41.1 intein system enables the toxin chains to self-assemble into the correct disulfide-bonded structure through protein trans-splicing. The intein-mediated reaction automatically forms the native disulfide connections between heavy and light chains without requiring external oxidizing agents or complex folding conditions.

Inventive Principle:
Principle #25Self-service

Solution Approach 2:

The invention changes the chemical parameters of the assembly process by using intein trans-splicing instead of traditional disulfide bond formation. This parameter change allows the reaction to proceed under milder, more controlled conditions with higher precision and fewer additional process steps.

Inventive Principle:
Principle #35Parameter changes

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 enables safe, high-yield production of botulinum toxin in a soluble form, simplifying the production process and avoiding the formation of inactive dimers, thus enhancing the toxin's activity and reducing production costs.

Implementation Method 1

screening for a suitable intein, and joining a heavy chain and a light chain of the botulinum toxin to each other using the intein

Methodology Applied
Scientific EffectProtein trans-splicing:

Implementation Method 2

forming a disulfide bond between them

Methodology Applied
Scientific EffectDisulfide bond formation:

Data Source

PatentEP4711379A1Method for producing botulinum neurotoxin using soluble partner and GP41.1 intein
Publication Date: 2026.03.18 MVRIX CO LTD
  • EP4711379A1 patent drawingFigure 1
  • EP4711379A1 patent drawingFigure 2(A)~2(B)
  • EP4711379A1 patent drawingFigure 3(A)~3(B)

AI summary

Disclosed is a method of producing botulinum toxin by a recombinant method using a soluble partner and GP41.1 intein. It was found that a heavy chain receptor binding domain (HC) of botulinum toxin with low solubility may be expressed in a soluble form in E. coli by linking a soluble partner. In addition, it was found that the soluble partner, which is generally a protein having a very large molecular weight, is easily removed using GP41.1 intein.