EN-apyrase Constructs for Extended Half-Life and Stability

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Solution Overview

Problem

Current apyrases have limitations in terms of stability, solubility, and half-life, which affect their therapeutic efficacy in treating thrombotic and inflammation-related diseases, and there is a need for improved pharmacokinetic properties and purification methods.

Innovation Solution

The development of enhanced apyrases (EN-apyrases) with improved pharmacokinetic properties, including longer half-life, higher stability, and solubility, achieved through specific design of expression vectors and culture conditions in Chinese hamster ovary cells, resulting in homogeneous preparations that can be readily purified.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If conventional apyrases are used, then thrombotic and inflammation-related diseases can be treated, but the therapeutic efficacy is limited due to short half-life and low stability

Engineering Contradiction:
Improvetherapeutic efficacyVSAvoidhalf-life
Core Design Contradiction:
ReliabilityVSDuration of action of stationary object

Solution Approach 1:

The patent modifies the amino acid sequence of CD39L3 by substituting arginine for glycine at residue 67 and threonine for arginine at residue 69. These parameter changes in the protein structure enhance ADPase activity and extend the half-life of the apyrase enzyme, directly resolving the contradiction between therapeutic efficacy and duration of action

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent creates a fusion protein by combining the modified CD39L3 enzyme with a carrier protein or peptide. This composite structure improves the stability and half-life of the apyrase while maintaining its therapeutic function, addressing both the reliability and duration limitations of conventional apyrases

Inventive Principle:
Principle #40Composite materials

2Reliability

If conventional apyrases are used, then anti-platelet activity is achieved, but solubility and stability are insufficient for effective therapy

Engineering Contradiction:
Improveanti-platelet activityVSAvoidsolubility
Core Design Contradiction:
ReliabilityVSStability of the object's composition

Solution Approach 1:

The amino acid substitutions at residues 67 and 69 not only enhance enzymatic activity but also improve the solubility and compositional stability of the apyrase protein. These parameter changes in the primary structure lead to improved folding, stability, and solubility characteristics essential for effective therapy

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

By fusing CD39L3 with appropriate carrier proteins or peptides, the patent creates composite structures that enhance solubility and stability while preserving anti-platelet activity. The fusion partners provide structural stability and improve pharmacokinetic properties

Inventive Principle:
Principle #40Composite materials

3Manufacturing precision

If purified apyrase preparations are produced, then therapeutic consistency is achieved, but purification is difficult due to heterogeneity

Engineering Contradiction:
Improvepreparation consistencyVSAvoidpurification difficulty
Core Design Contradiction:
Manufacturing precisionVSEase of manufacture

Solution Approach 1:

The patent produces homogeneous preparations of modified CD39L3 apyrase through optimized expression systems and purification methods. The amino acid modifications and fusion protein design result in more homogeneous protein populations that are easier to purify to high consistency, directly addressing the contradiction between manufacturing precision and ease of manufacture

Inventive Principle:
Principle #33Homogeneity

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

EN-apyrases exhibit extended circulating half-life, enhanced glycosylation, and improved therapeutic efficacy as anti-platelet and anti-inflammatory agents, providing effective treatment for conditions such as thrombosis and inflammation with prolonged protective effects.

Implementation Method 1

the expression of the fusion polypeptide in CHO cells which secretes both of the components of the fusion polypeptide upon cleavage

Methodology Applied
Scientific EffectSecretion:

Implementation Method 2

upon cleavage of the alpha-lactalbumin signal peptide at its carboxyl-terminus from the amino terminus of the carboxyl-proximal fusion polypeptide

Methodology Applied
Scientific EffectProteolytic cleavage:

Implementation Method 3

Apyrases (Ecto-ATP diphosphohydrolases) constitute a group of enzymes catalyzing metabolism of ATP to ADP and ADP to AMP

Methodology Applied
Scientific EffectEnzymatic hydrolysis: Hydrolysis

Data Source

PatentEP2523971B1Therapeutic apyrase constructs, apyrase agents, and production methods
Publication Date: 2015.02.25 APT THERAPEUTICS INC
  • EP2523971B1 patent drawingFigure 1
  • EP2523971B1 patent drawingFigure 2
  • EP2523971B1 patent drawingFigure 3~4

AI summary

This invention provides a new class of enhanced apyrases (EN-apyrases) with superior pharmacokinetic, pharmacodynamic, and pharmacochemical properties and which can be purified using simplified procedures. The invention further provides constructs for transforming a cell to produce these EN-apyrases. The EN-apyrase construct comprises sequences encoding a signal sequence, a linker, and a soluble apyrase. Also provided are preparations of apyrases and methods for producing apyrase in culture cells and purification thereof.