A1PI Ultrafiltration Formulation for High-Concentration Stability

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Solution Overview

Problem

Current methods for producing Alpha-1 Proteinase Inhibitor (A1PI) result in low concentrations, requiring burdensome intravenous administration and causing hypersensitive reactions, limiting its suitability for self-administration and increasing treatment costs.

Innovation Solution

Conventional tangential flow filtration (TFF) ultrafiltration is used to concentrate A1PI to at least 110 mg/mL without buffers or salts, followed by formulation with uncharged excipients like sorbitol, trehalose, or alanine to achieve stable concentrations between 100 mg/mL and 200 mg/mL, suitable for various parenteral routes.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Quantity of substance

If conventional TFF ultrafiltration is used to concentrate A1PI, then concentration increases, but aggregate formation increases making the product unacceptable

Engineering Contradiction:
ImproveA1PI concentrationVSAvoidproduct acceptability
Core Design Contradiction:
Quantity of substanceVSReliability

Solution Approach 1:

The patent changes the chemical composition parameters by formulating with uncharged excipients (sorbitol, trehalose, alanine, sucrose, or mannitol) instead of conventional buffers and salts. This parameter change allows achieving A1PI concentrations ≥110 mg/mL through TFF ultrafiltration without forming unacceptable levels of aggregates, thereby resolving the contradiction between concentration increase and product acceptability.

Inventive Principle:
Principle #35Parameter changes

2Stability of the object's composition

If buffers and salts are used to maintain pH during concentration, then pH stability is maintained, but aggregate formation increases

Engineering Contradiction:
ImprovepH stabilityVSAvoidaggregate level
Core Design Contradiction:
Stability of the object's compositionVSQuantity of substance

Solution Approach 1:

The patent replaces charged buffer and salt systems with uncharged excipients that can still maintain pH stability through different mechanisms. This parameter change eliminates the harmful interaction between charged excipients and A1PI that causes aggregate formation, while maintaining the necessary pH stability for product quality.

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent uses simple uncharged molecules (sorbitol, trehalose, alanine, sucrose, mannitol) as excipients instead of complex buffer systems. These simple substances effectively maintain pH stability without causing aggregate formation, resolving the contradiction between pH stability and aggregate reduction.

Inventive Principle:
Principle #27Cheap short-living objects (Disposable)

3Quantity of substance

If further concentration steps (SPTFF) are performed to achieve >200 mg/mL, then concentration increases, but process complexity increases

Engineering Contradiction:
ImproveA1PI concentrationVSAvoidprocess steps
Core Design Contradiction:
Quantity of substanceVSDevice complexity

Solution Approach 1:

By changing the excipient parameter to uncharged molecules, the patent enables achieving therapeutic concentrations (≥110 mg/mL) in a single TFF ultrafiltration step without requiring additional SPTFF processing steps. This parameter change simplifies the overall process while maintaining high concentration levels.

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 method enables easier self-administration of A1PI, reduces treatment costs, and provides stable, highly concentrated formulations suitable for intravenous, subcutaneous, and intradermal use, overcoming previous limitations of low yield and hypersensitivity.

Implementation Method 1

conventional TFF ultrafiltration is used to concentrate A1PI to at least 110 mg/mL

Methodology Applied
Scientific EffectUltrafiltration: Semipermeable Membrane

Data Source

PatentUS12479907B2Method for obtaining Alpha-1 proteinase inhibitor
Publication Date: 2025.11.25 GRIFOLS WORLDWIDE OPERATIONS

AI summary

A method for obtaining Alpha-1 Proteinase Inhibitor for obtaining highly-concentrated Alpha-1 Proteinase Inhibitor (A1PI) includes preparing a solution of A1PI by concentrating an initial solution of A1PI by tangential flow filtration (TFF) against water. The final concentration of A1PI in the concentrated solution is at least 110 mg/ml. The TFF can be carried out against water for injection (WFD).