rAAV Pharmaceutical Composition Stabilization

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Solution Overview

Problem

The stability of recombinant adeno-associated virus (rAAV) pharmaceutical compositions is a concern due to potential environmental changes and manufacturing challenges, which can affect the safety and efficacy of the product.

Innovation Solution

A pharmaceutical composition comprising recombinant adeno-associated virus (AAV) is formulated with a buffer, an amino acid, a stabilizer, and a nonionic surfactant, specifically citrate buffer, aspartic acid or proline as amino acids, sucrose or trehalose as stabilizers, and poloxamer 188 as a nonionic surfactant, to enhance stability.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Ease of manufacture

If recombinant adeno-associated virus (rAAV) pharmaceutical composition is subjected to environmental changes and manufacturing processes, then the virus can be produced and processed, but the stability and quality of the product deteriorates

Engineering Contradiction:
Improvemanufacturing processabilityVSAvoidproduct stability
Core Design Contradiction:
Ease of manufactureVSStability of the object's composition

Solution Approach 1:

The patent applies preliminary action by pre-formulating the rAAV composition with stabilizing agents (sugars, amino acids, surfactants, buffers) before manufacturing and storage processes. This pre-protection strategy ensures the virus is already shielded against environmental stresses during subsequent manufacturing, transportation, and storage operations, thereby maintaining product quality without compromising manufacturability

Inventive Principle:
Principle #10Preliminary action

Solution Approach 2:

The patent uses intermediary substances (stabilizing agents including sugars like sucrose and trehalose, amino acids like aspartic acid and proline, nonionic surfactants like poloxamer 188, and buffers like citrate) as mediators between the rAAV virus and environmental stressors. These intermediaries form a protective matrix that buffers the virus against temperature changes, mechanical stress, and chemical degradation during manufacturing and storage

Inventive Principle:
Principle #24Intermediary (Mediator)

2Duration of action of stationary object

If storage time is extended to ensure adequate supply, then product availability improves, but virus activity and purity deteriorate

Engineering Contradiction:
Improvestorage durationVSAvoidvirus activity and purity
Core Design Contradiction:
Duration of action of stationary objectVSReliability

Solution Approach 1:

The patent implements beforehand cushioning by incorporating multiple stabilizing agents that create a protective environment for the rAAV virus before storage begins. The combination of sugars (sucrose, trehalose), amino acids (aspartic acid, proline), surfactants (poloxamer 188), and buffers (citrate) provides cumulative protection against degradation mechanisms, allowing the virus to maintain its activity and purity over extended storage periods of months to years

Inventive Principle:
Principle #11Beforehand cushioning (Prior cushioning)

Solution Approach 2:

The patent employs composite materials by formulating a complex stabilization system comprising multiple types of agents: carbohydrates (sucrose, trehalose, mannitol), amino acids (aspartic acid, proline, arginine), nonionic surfactants (poloxamer 188, polysorbate 80), and buffers (sodium citrate). This composite formulation works synergistically to protect the rAAV virus from various degradation pathways simultaneously, enabling long-term stable storage while maintaining viral reliability

Inventive Principle:
Principle #40Composite materials

3Stability of the object's composition

If temperature control is maintained to preserve virus stability, then product quality is maintained, but manufacturing and distribution complexity increases

Engineering Contradiction:
Improvevirus stabilityVSAvoidstorage and distribution system complexity
Core Design Contradiction:
Stability of the object's compositionVSDevice complexity

Solution Approach 1:

The patent applies this principle by using small molecules (sugars, amino acids, surfactants, buffers) that are chemically stable, inexpensive, and do not require complex preservation themselves. These small molecular stabilizers create a protective environment that allows the rAAV virus to be stored and distributed without expensive refrigeration infrastructure, effectively replacing complex temperature control systems with simple chemical stabilization

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

Data Source

PatentEP4537856A1Adeno-associated virus pharmaceutical composition and use thereof
Publication Date: 2025.04.16 CHENGDU ORIGEN BIOTECHNOLOGY CO LTD
  • EP4537856A1 patent drawingFigure 1A~1B
  • EP4537856A1 patent drawingFigure 2
  • EP4537856A1 patent drawing

AI summary

Provided is a pharmaceutical composition, the pharmaceutical composition comprising a recombinant adeno-associated virus (AAV), a buffer solution, an amino acid, a stabilizer, and a nonionic surfactant. The pharmaceutical composition has good stability.