Recombinant Factor VIII Production via Pichia pastoris Segmentation

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Solution Overview

Problem

Current methods for producing recombinant Factor VIII protein face challenges such as scarcity of plasma-derived sources, risk of viral contamination, and difficulty in expressing full-length recombinant Factor VIII, which are addressed by using the Pichia pastoris expression system to generate functional Factor VIII chains.

Innovation Solution

The process involves introducing polynucleotide sequences encoding the heavy and light chains of Factor VIII into Pichia pastoris, expressing and purifying these chains using IMAC and HLAC, and reconstituting them in the presence of divalent metal ions to produce a functional recombinant Factor VIII protein with improved activity.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Quantity of substance

If plasma-derived sources are used to produce Factor VIII protein, then the protein can be obtained through traditional purification methods, but the scarcity of plasma from healthy donors limits the quantity available for purification

Engineering Contradiction:
Improvequantity of Factor VIII proteinVSAvoidproduction capacity
Core Design Contradiction:
Quantity of substanceVSProductivity

Solution Approach 1:

The Factor VIII protein is divided into two separate chains (heavy chain and light chain) that are expressed independently in Pichia pastoris. This segmentation allows each chain to be produced separately and then reconstituted, overcoming the limitation of plasma scarcity by enabling scalable microbial expression of individual chains rather than relying on limited plasma sources.

Inventive Principle:
Principle #1Segmentation

Solution Approach 2:

Instead of purifying Factor VIII from natural plasma sources, the patent creates recombinant copies of the heavy and light chains using the Pichia pastoris expression system. This copying approach allows unlimited production of Factor VIII chains through microbial fermentation, independent of plasma donor availability.

Inventive Principle:
Principle #26Copying

2Reliability

If plasma-derived Factor VIII is purified, then treatment for Haemophilia A can be provided, but there is a risk of viral contamination

Engineering Contradiction:
Improvesafety of Factor VIII treatmentVSAvoidviral contamination risk
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent produces recombinant Factor VIII chains in Pichia pastoris, creating a viral-free copy of the therapeutic protein. Since Pichia pastoris is a yeast system, it does not carry human viruses, thereby eliminating the viral contamination risk associated with plasma-derived products while providing the same therapeutic function.

Inventive Principle:
Principle #26Copying

Solution Approach 2:

The Pichia pastoris expression system serves as an intermediary host that produces Factor VIII chains without introducing viral contaminants. This intermediary microbial system allows safe production of human Factor VIII by separating the production process from human plasma sources.

Inventive Principle:
Principle #24Intermediary (Mediator)

3Reliability

If full-length recombinant Factor VIII is expressed, then complete Factor VIII activity can be achieved, but the expression is difficult due to the large size and complex structure

Engineering Contradiction:
ImproveFactor VIII activityVSAvoidexpression difficulty
Core Design Contradiction:
ReliabilityVSEase of manufacture

Solution Approach 1:

The full-length Factor VIII protein is segmented into heavy chain and light chain components that are expressed separately in Pichia pastoris. This segmentation reduces the complexity of expression for each individual chain, making manufacturing easier while maintaining the ability to reconstitute the complete functional protein.

Inventive Principle:
Principle #1Segmentation

Solution Approach 2:

The heavy and light chains are expressed and purified separately as preliminary steps before final reconstitution. This preliminary separation simplifies the expression process by avoiding the need to handle the complete complex protein structure during production, while ensuring both chains are available for assembly into the active heterodimer.

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

This method results in a recombinant Factor VIII protein with enhanced activity, overcoming the limitations of traditional production methods and providing a safer, more efficient alternative for treating Haemophilia A.

Implementation Method 1

introducing polynucleotide sequences encoding the heavy and light chains of Factor VIII into Pichia pastoris, expressing and purifying these chains

Methodology Applied
Scientific EffectGenetic expression:

Implementation Method 2

purifying these chains using IMAC and HLAC

Methodology Applied
Scientific EffectAffinity chromatography: Chromatography

Implementation Method 3

purifying these chains using IMAC and HLAC

Methodology Applied
Scientific EffectAffinity chromatography: Chromatography

Implementation Method 4

reconstituting them in the presence of divalent metal ions to produce a functional recombinant Factor VIII protein

Methodology Applied
Scientific EffectMetal ion coordination:

Data Source

PatentEP2895199B1Methods for producing recombinant factor viii chains from non-filamentous fungi, their functional reconstitution and applications thereof
Publication Date: 2019.08.21 CENT FOR BIOSEPARATION TECH - VIT
  • EP2895199B1 patent drawingFigure 1A~1B
  • EP2895199B1 patent drawingFigure 2~3B
  • EP2895199B1 patent drawingFigure 4A~4D

AI summary

The present disclosure relates specifically to a process of producing heavy chain peptide and/or light chain peptide of recombinant Factor VIII protein using Pichia pastoris expression system. The disclosure further relates to a process of producing a functional recombinant Factor VIII protein by reconstituting the Heavy chain and Light chain produced using said Pichia pastoris expression system. The said functional recombinant Factor VIII protein shows improved activity and therefore is used in the management of haemophilia.