Thiol-Functionalising Reagents for Site-Specific Protein PEGylation

Resolve Bottlenecks,
Find Innovative Solutions
Generate Solutions

Solution Overview

Problem

Current pegylation methods for modifying proteins are uncontrolled, leading to heterogeneous products due to the use of reactive cross-linkers like maleimide, which react unpredictably with multiple functional groups, and glycosylation is rarely achieved in prokaryotic hosts, affecting the properties and stability of therapeutic polypeptides.

Innovation Solution

The use of nitrogen-containing heterocyclic aromatic rings with vinyl substituents covalently linked to poly(alkylene glycol) molecules or glycan groups, which react specifically with thiol groups in proteins to form stable thioether bonds, allowing for controlled modification and site-specific glycosylation.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If reactive cross-linkers like maleimide are used for pegylation, then the polypeptide can be modified to improve stability and half-life, but the reaction becomes uncontrolled producing heterogeneous products

Engineering Contradiction:
ImprovestabilityVSAvoidproduct homogeneity
Core Design Contradiction:
ReliabilityVSManufacturing precision

Solution Approach 1:

The patent introduces a site-specific thiol group as an intermediary target for PEGylation. Instead of allowing the cross-linker to react randomly with multiple functional groups, the thiol group serves as a specific mediator that directs the PEG attachment to a predetermined location on the polypeptide, ensuring both stability improvement and product homogeneity

Inventive Principle:
Principle #24Intermediary (Mediator)

Solution Approach 2:

The invention applies local quality by creating a specific reactive site (thiol group) at a particular location on the polypeptide chain. This localized reactive group ensures that PEGylation occurs only at the desired position rather than throughout the entire molecule, producing homogeneous products with consistent pharmacokinetic properties

Inventive Principle:
Principle #3Local quality

2Adaptability or versatility

If PEG molecules react with multiple functional groups in polypeptides, then more extensive modification can be achieved, but the resulting products have variable structures and properties

Engineering Contradiction:
Improvemodification extentVSAvoidstructural consistency
Core Design Contradiction:
Adaptability or versatilityVSManufacturing precision

Solution Approach 1:

The patent extracts the reactivity from multiple functional groups and concentrates it in a single thiol group. By introducing or utilizing one specific cysteine residue with a thiol group, the method takes out the indiscriminate reactivity of multiple groups and channels all PEGylation activity through this single controlled site, achieving both extensive modification and structural consistency

Inventive Principle:
Principle #2Taking out (Extraction)

3Manufacturing precision

If protecting groups are used to control reaction site, then site-specific modification can be achieved, but the process becomes complex and difficult to implement in prokaryotic systems

Engineering Contradiction:
Improvesite specificityVSAvoidprocess complexity
Core Design Contradiction:
Manufacturing precisionVSDevice complexity

Solution Approach 1:

The patent employs self-service by utilizing the polypeptide's own thiol group (either naturally present or introduced via site-directed mutagenesis) as the reactive site for PEGylation. This eliminates the need for external protecting groups and complex multi-step protection/deprotection sequences, significantly simplifying the process while maintaining high site specificity, especially in prokaryotic expression systems

Inventive Principle:
Principle #25Self-service

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

This approach enables precise and controlled modification of proteins, improving their stability, biological half-life, and immunological properties, while allowing for glycosylation in prokaryotic hosts, resulting in more consistent and effective therapeutic polypeptide products.

Implementation Method 1

the vinyl substituent of the functionalising reagent reacts with the thiol group of the polypeptide, thereby to covalently link the poly(alkylene glycol) molecule to the polypeptide

Methodology Applied
Scientific EffectThioether bond formation: Chemical Bonding

Data Source

PatentEP2886132B1Thiol-functionalising reagents and their uses
Publication Date: 2018.04.04 IKSUDA THERAPEUTICS LTD
  • EP2886132B1 patent drawingFigure 1~2
  • EP2886132B1 patent drawingFigure 3~4
  • EP2886132B1 patent drawingFigure 5A~6

AI summary

Reagents and methods for functionalising polypeptides with moieties poly(alkylene glycol) molecules and glycan groups are disclosed that are based on a functionalising reagent which comprises a nitrogen containing heterocyclic aromatic ring having a vinyl substituent that is capable of reacting with one or more thiol groups that are naturally present, or have been introduced into, the polypeptide, for example by employing a thiol group of one or more cysteine residues. The functionalising reagent is covalently linked to a poly(alkylene glycol) molecule, such as a polyethylene glycol (PEG) molecule, or a glycan group so that the reaction between the vinyl group and the thiol group in the polypeptide covalently links the polypeptide to the poly(alkylene glycol) molecule and/or the glycan group.