Tetrapeptide Linkers for Rapid Amine Liberation

Resolve Bottlenecks,
Find Innovative Solutions
Generate Solutions

Solution Overview

Problem

Current peptide proteolyzable pro-drugs do not liberate primary amine constituents at a rate comparable to self-immolating PABC type analogues, and there is concern about toxicity from aza-quinone methide generated during PABC elimination, which can react with N, O, and S-nucleophiles.

Innovation Solution

Development of tetrapeptide linkers comprising L-phenylalanine, L-citrulline, L-phenylalanine, and L-proline sequences that are cleaved by proteolytic enzymes, allowing for rapid liberation of amine-containing compounds, and the use of amine-reactive groups to form stable amide bonds, reducing toxicity concerns.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Productivity

If self-immolating PABC spacer is used for controlled drug release, then the primary amine constituent is liberated rapidly, but toxicity increases due to aza-quinone methide generation

Engineering Contradiction:
Improverate of amine liberationVSAvoidtoxicity from aza-quinone methide
Core Design Contradiction:
ProductivityVSObject-generated harmful factors

Solution Approach 1:

The invention extracts and removes the harmful aza-quinone methide self-immolative mechanism from the PABC spacer, retaining only the beneficial proteolytic cleavage function. The new tetrapeptide linker eliminates the quinonimine methide generation step while maintaining rapid amine release through direct protease cleavage of the peptide bond.

Inventive Principle:
Principle #2Taking out (Extraction)

Solution Approach 2:

The invention converts the harmful self-immolative elimination mechanism into a beneficial direct proteolytic cleavage mechanism. By using protease-cleavable peptide sequences, the system achieves rapid amine liberation without generating toxic intermediates, effectively transforming the harmful chemical pathway into a safe biological degradation pathway.

Inventive Principle:
Principle #22Blessing in disguise (Convert harm into benefit)

2Stability of the object's composition

If peptide proteolyzable pro-drugs are used, then stability is improved, but the rate of amine liberation is slower compared to PABC type analogues

Engineering Contradiction:
Improvelinker stabilityVSAvoidrate of amine liberation
Core Design Contradiction:
Stability of the object's compositionVSProductivity

Solution Approach 1:

The invention changes the kinetic parameters of proteolytic cleavage by selecting specific peptide sequences (such as L-phenylalanine-L-citrulline-L-phenylalanine-L-proline) that are highly susceptible to protease attack. This parameter optimization maintains linker stability in circulation while enabling rapid cleavage and amine release at the target site, resolving the contradiction between stability and liberation rate.

Inventive Principle:
Principle #35Parameter changes

3Ease of operation

If aza-quinone methide is generated during PABC elimination, then spontaneous release is achieved, but harmful reactions with N, O, and S-nucleophiles occur

Engineering Contradiction:
Improvespontaneous release capabilityVSAvoidreaction with nucleophiles
Core Design Contradiction:
Ease of operationVSObject-affected harmful factors

Solution Approach 1:

The invention introduces proteases as intermediary enzymes that mediate the cleavage process. Instead of spontaneous self-immolative elimination that generates harmful intermediates, the system uses protease-mediated hydrolysis of peptide bonds as a safe intermediary step, which directly releases the amine without generating reactive quinonimine methide species that would react with nucleophiles.

Inventive Principle:
Principle #24Intermediary (Mediator)

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 tetrapeptide linkers exhibit improved kinetics of cleavage and stability, efficiently releasing the amine-containing compounds while minimizing toxicity, providing a more effective and safer approach for drug delivery.

Implementation Method 1

In the presence of proteolytic enzymes, hydrolysis of the anilide bond triggers a 1,6-elimination cascade

Methodology Applied
Scientific EffectProteolysis: Decomposition (biological)

Implementation Method 2

forming stable amide bonds

Methodology Applied
Scientific EffectCovalent bonding: Chemical Bonding

Data Source

PatentEP3302516B1Biologically cleavable tetrapeptide linking agents
Publication Date: 2020.12.30 ARROWHEAD PHARMACEUTICALS INC
  • EP3302516B1 patent drawingFigure 1
  • EP3302516B1 patent drawingFigure 2A~2B
  • EP3302516B1 patent drawingFigure 3

AI summary

Tetrapeptide linkers for reversibly linking a first compound to a amine-containing second compound are described. Compounds containing the tetrapeptide linkers and methods of using the tetrapeptide linkers are also described.