Heparin-Binding Peptide Conjugates for Targeted Drug Delivery

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Solution Overview

Problem

Current methods for delivering therapeutic agents, such as recombinant cytokines and small molecules, often result in off-target effects due to systemic administration and non-specific activity, necessitating targeted delivery to specific tissues like cartilage, brain, and spinal cord.

Innovation Solution

The use of novel heparin-binding peptides (HB) fused or conjugated to therapeutic proteins or small molecules, which selectively bind to proteoglycans in target tissues, enabling targeted delivery through conjugation or indirect linkage, utilizing linker peptides for enhanced specificity.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If therapeutic agents are administered systemically, then broad coverage and availability to multiple tissues is achieved, but off-target effects and reduced specificity occur

Engineering Contradiction:
Improvespecificity of therapeutic deliveryVSAvoidbroad tissue coverage
Core Design Contradiction:
ReliabilityVSAdaptability or versatility

Solution Approach 1:

The patent applies local quality by modifying the therapeutic agent to have different properties at different locations: the HB peptide confers heparin-binding capability specifically at target sites expressing proteoglycans, while the therapeutic payload remains inactive or less active elsewhere. This creates spatially heterogeneous functionality that achieves both specificity and controlled broad coverage.

Inventive Principle:
Principle #3Local quality

Solution Approach 2:

The heparin-binding peptide acts as an intermediary that mediates between the therapeutic agent and target tissues. The HB peptide binds to heparan sulfate proteoglycans on target cells, serving as a bridge that directs the therapeutic agent to specific locations while preventing non-specific distribution throughout the body.

Inventive Principle:
Principle #24Intermediary (Mediator)

2Duration of action of moving object

If therapeutic agents are delivered non-specifically, then ease of administration is maintained, but retention time and activity in target tissues decrease

Engineering Contradiction:
Improveretention time in target tissueVSAvoidsimplicity of administration
Core Design Contradiction:
Duration of action of moving objectVSEase of operation

Solution Approach 1:

The patent merges two functional components into a single therapeutic construct: the heparin-binding peptide is fused or conjugated to the therapeutic agent, creating a unified molecule that combines targeting functionality with therapeutic activity. This eliminates the need for separate administration steps while achieving prolonged retention.

Inventive Principle:
Principle #5Merging (Combining)

Solution Approach 2:

The HB peptide is pre-attached to the therapeutic agent before administration, enabling the conjugate to self-target upon reaching the body. The targeting functionality is prepared in advance, so no additional targeting steps are needed during or after administration, maintaining simplicity while enhancing retention.

Inventive Principle:
Principle #10Preliminary action

3Reliability

If heparin-binding peptides are conjugated to therapeutic agents, then targeted delivery to proteoglycan-expressing tissues is achieved, but conjugation complexity increases

Engineering Contradiction:
Improvetargeted delivery efficiencyVSAvoidconjugation process complexity
Core Design Contradiction:
ReliabilityVSDevice complexity

Solution Approach 1:

The HB peptide-conjugated therapeutic agents are designed to self-assemble or self-target through inherent biochemical properties. The heparin-binding capability is intrinsic to the HB peptide sequence, allowing the conjugate to automatically find and bind to proteoglycan-expressing tissues without requiring complex external guidance systems or multi-step conjugation procedures.

Inventive Principle:
Principle #25Self-service

Solution Approach 2:

The patent utilizes changes in biochemical parameters (positive charge density, heparin affinity) of the HB peptide to achieve targeted delivery. By optimizing the amino acid sequence of HB to enhance heparin binding, the system achieves high targeted delivery efficiency through intrinsic molecular properties rather than complex structural designs or multiple components.

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

This approach allows for selective and sustained delivery of therapeutic agents to proteoglycan-expressing tissues, reducing systemic circulation and enhancing retention and activity in target areas, as demonstrated by prolonged retention and efficacy in cartilage and spinal cord tissues.

Implementation Method 1

the HB portion of the composition is positively charged through many lysine and arginine residues in the HB peptide, which binds to cellular or tissue expressing proteoglycans which are negatively charged by sulfate groups

Methodology Applied
Scientific EffectElectrostatic interaction: Coulomb's Law

Data Source

PatentEP2864360B1Targeted therapeutics
Publication Date: 2017.09.06 THE BRIGHAM & WOMEN S HOSPITAL INC
  • EP2864360B1 patent drawingFigure 1~2
  • EP2864360B1 patent drawingFigure 3~4
  • EP2864360B1 patent drawingFigure 5~6

AI summary

The present invention relates to compositions, methods and kits for delivery of active agents (X) to cells or tissues that express proteoglycans. More specifically, the present invention relates to conjugates comprising (HB-linker)n-Xm-(linker-HB)o, where HB is a heparin binding protein, X is an active agents such as a therapeutic protein (including a functional fragment or variant thereof), or a therapeutic small molecule, linker is a linker entity and m is an integer of at least 1, and n+o is an integer of at least 1. Other aspects relate to use of a HB-X conjugate in methods to deliver active agents to cartilage for the treatment of cartilage related disease and disorders, the use of the HB-X conjugate in methods to deliver active agents to neuronal tissues (e.g., brain and spinal cord) for the treatment of neurological disorders, and the use of the HB-X conjugates in methods for the treatment of eye conditions and disorders and inflammatory conditions and disorders.