Blood Brain Barrier Shuttle Peptide Antibody Delivery

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Solution Overview

Problem

The blood-brain barrier (BBB) and blood-CSF barrier pose significant challenges for the delivery of therapeutic and diagnostic agents to the brain, as they restrict the penetration of large biotherapeutic drugs and small molecules, limiting their therapeutic effect due to low brain uptake and accumulation within brain endothelial cells, leading to degradation.

Innovation Solution

A blood-brain barrier shuttle comprising a brain effector entity, such as a full-length antibody, covalently coupled with a brain-targeting peptide motif, specifically designed to enhance delivery across the BBB, utilizing a pharmaceutical formulation that includes a pharmaceutical carrier for targeted delivery to the brain, particularly for neurodegenerative disorders like Alzheimer's disease and Multiple Sclerosis.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Quantity of substance

If monoclonal antibodies or other biotherapeutics are administered systemically, then therapeutic potential for CNS pathology is achieved, but brain penetration is strictly limited by the blood-brain barrier resulting in only approximately 0.1% penetration into the CNS compartment

Engineering Contradiction:
Improvebrain uptake of therapeutic agentVSAvoidblood-brain barrier obstruction
Core Design Contradiction:
Quantity of substanceVSObject-affected harmful factors

Solution Approach 1:

The patent uses a brain-targeting peptide as an intermediary molecule that facilitates the transport of therapeutic agents across the blood-brain barrier. The peptide acts as a mediator that binds to the therapeutic agent and enables its passage through the BBB via receptor-mediated transcytosis, thereby resolving the contradiction between systemic administration and brain penetration limitation

Inventive Principle:
Principle #24Intermediary (Mediator)

Solution Approach 2:

The invention creates a composite structure by covalently coupling the brain-targeting peptide with the therapeutic agent (antibody or small molecule). This composite shuttle molecule combines the brain-penetrating capability of the peptide with the therapeutic function of the agent, enabling effective CNS delivery while overcoming the BBB obstruction

Inventive Principle:
Principle #40Composite materials

2Reliability

If therapeutic agents are delivered to the brain, then CNS therapeutic effect is achieved, but missorting and accumulation within organelles in brain endothelial cells leads to degradation of the biotherapeutic

Engineering Contradiction:
Improvetherapeutic effect in CNSVSAvoiddegradation of biotherapeutic in brain endothelial cells
Core Design Contradiction:
ReliabilityVSLoss of substance

Solution Approach 1:

The brain-targeting peptide enables the therapeutic agent to rapidly traverse the brain endothelial cells via transcytosis, minimizing the time the agent spends within the cells. This rapid transit reduces the opportunity for missorting and accumulation in degradative organelles, thereby preserving the therapeutic agent's integrity and activity

Inventive Principle:
Principle #21Skipping (Rushing through)

Solution Approach 2:

The peptide is designed with specific amino acid motifs that pre-program the transcytosis pathway, ensuring the therapeutic agent is directed through the correct cellular route before degradation can occur. The preliminary structural design of the peptide prevents off-pathway routing that would lead to degradation

Inventive Principle:
Principle #10Preliminary action

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 shuttle effectively increases the brain uptake of therapeutic agents, reducing missorting and degradation within brain endothelial cells, thereby enhancing the therapeutic effect and concentration of drugs in the central nervous system.

Implementation Method 1

utilize transcytosis pathways mediated by endogenous receptors expressed on the brain capillary endothelium

Methodology Applied
Scientific EffectTranscytosis:

Implementation Method 2

a blood brain barrier shuttle comprising a brain effector entity and a brain targeting peptide

Methodology Applied
Scientific EffectCovalent bonding: Chemical Bonding

Data Source

PatentEP3107928B1Blood brain barrier shuttle
Publication Date: 2019.09.04 F HOFFMANN LA ROCHE & CO AG
  • EP3107928B1 patent drawingFigure 1A~1B
  • EP3107928B1 patent drawingFigure 2A~2D
  • EP3107928B1 patent drawingFigure 3

AI summary

The invention provides a blood brain barrier shuttle comprising a brain effector entity and a brain targeting peptide.