Macrocyclic Peptide Pseudo Bicyclic Structure for Cell Penetration

Resolve Bottlenecks,
Find Innovative Solutions
Generate Solutions

Solution Overview

Problem

Peptide drugs face challenges in penetrating cell membranes and maintaining stability due to their chemical and biological properties, leading to limited effectiveness in reaching intracellular targets and resistance to protease degradation.

Innovation Solution

Development of macrocyclic peptides with a pseudo bicyclic structure formed by noncovalent interaction of hydrophobic side chains, which enhances stability in hydrophilic environments and allows for cell membrane penetration by exposing hydrophobic groups in hydrophobic environments, combined with a library screening method to identify effective inhibitors.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If peptide drugs are used to achieve high affinity and specificity for target molecules, then physiological activity is improved, but cell membrane penetration ability deteriorates

Engineering Contradiction:
ImprovespecificityVSAvoidcell membrane penetration
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The peptide structure is designed to be dynamic, allowing it to change conformation between a compact state (for membrane penetration) and an extended state (for target binding). The peptide can dynamically adjust its structure to overcome the contradiction between maintaining specificity and achieving membrane penetration.

Inventive Principle:
Principle #15Dynamics

Solution Approach 2:

The invention changes key parameters of the peptide including incorporating non-natural amino acids with hydrophobic side chains, modifying charge distribution, and adjusting hydrophobicity/hydrophilicity balance. These parameter changes enable the peptide to both penetrate membranes and maintain high affinity for targets.

Inventive Principle:
Principle #35Parameter changes

2Reliability

If peptide drugs are used to achieve high specificity for target molecules, then physiological activity is improved, but protease resistance deteriorates

Engineering Contradiction:
ImprovespecificityVSAvoidprotease resistance
Core Design Contradiction:
ReliabilityVSStability of the object's composition

Solution Approach 1:

The peptide is designed as a composite structure incorporating both natural amino acids (for target recognition and binding) and non-natural amino acids (for enhanced protease resistance). This composite approach allows the peptide to maintain specificity while gaining stability against proteolytic degradation.

Inventive Principle:
Principle #40Composite materials

Solution Approach 2:

Specific regions of the peptide are selectively modified with non-natural amino acids to provide localized protease resistance, while other regions maintain their natural structure for optimal target interaction. This local quality modification preserves specificity while enhancing stability.

Inventive Principle:
Principle #3Local quality

3Stability of the object's composition

If macrocyclization is applied to improve protease resistance and stability, then in vivo stability is improved, but cell membrane penetration ability deteriorates

Engineering Contradiction:
Improvein vivo stabilityVSAvoidcell membrane penetration
Core Design Contradiction:
Stability of the object's compositionVSObject-affected harmful factors

Solution Approach 1:

The macrocyclic peptide is designed with dynamic flexibility, allowing it to adopt different conformations. In hydrophilic environments, it maintains a compact macrocyclic structure for stability, while in hydrophobic environments (cell membranes), it can dynamically open or adjust to facilitate penetration.

Inventive Principle:
Principle #15Dynamics

Solution Approach 2:

The invention modifies the macrocyclic structure by incorporating amino acids with hydrophobic side chains and adjusting the overall hydrophobicity parameter. This allows the macrocycle to balance stability in aqueous environments with sufficient membrane permeability.

Inventive Principle:
Principle #35Parameter changes

4Object-affected harmful factors

If hydrophobic side chains are introduced to improve membrane penetration, then cell membrane permeability is improved, but in vivo stability deteriorates

Engineering Contradiction:
Improvecell membrane penetrationVSAvoidin vivo stability
Core Design Contradiction:
Object-affected harmful factorsVSStability of the object's composition

Solution Approach 1:

Hydrophobic side chains are strategically positioned at specific locations on the peptide structure (particularly in hydrophobic patches) to enable membrane interaction, while other regions maintain hydrophilic or polar characteristics for stability in aqueous physiological environments. This spatial differentiation of properties resolves the contradiction.

Inventive Principle:
Principle #3Local quality

Solution Approach 2:

The peptide employs a composite amino acid composition combining hydrophobic non-natural amino acids (for membrane penetration) with polar or charged amino acids (for in vivo stability). This composite composition allows simultaneous achievement of both membrane permeability and physiological stability.

Inventive Principle:
Principle #40Composite materials

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 macrocyclic peptides achieve enhanced in vivo stability and cell membrane permeability, enabling high affinity and specificity for intracellular targets, and efficient protein-protein interaction inhibition.

Implementation Method 1

a macrocyclic peptide having, in a ring portion thereof, an amino acid having at least two hydrophobic side chains has, in a hydrophilic environment, a pseudo bicyclic like structure through a noncovalent bond due to interaction between the hydrophobic side chains inside the ring

Methodology Applied
Scientific EffectHydrophobic interaction: Hydrophobe

Implementation Method 2

the macrocyclic peptide can acquire affinity for a hydrophobic environment because due to a dynamic change in the structure in the hydrophobic environment, the hydrophobic group is exposed outside the molecule

Methodology Applied
Scientific EffectHydrophobic interaction: Hydrophobe

Data Source

PatentEP3040417B1Macrocyclic peptide, method for producing same, and screening method using macrocyclic peptide library
Publication Date: 2023.12.13 THE UNIV OF TOKYO
  • EP3040417B1 patent drawingFigure 1A~1C
  • EP3040417B1 patent drawingFigure 2
  • EP3040417B1 patent drawingFigure 3

AI summary

An object of the present invention is to provide a peptide excellent in resistance against metabolism, having a stable structure in vivo, and capable of penetrating a cell membrane and reaching in cells. The present invention provides a macrocyclic peptide having a macrocyclic structure comprised of four or more amino acids. At least two amino acids not adjacent to each other have a hydrophobic side chain and the hydrophobic side chains interact with each other inside the ring of the macrocyclic peptide in a hydrophilic environment.