IsoTag^™AAV: an innovative, scalable & non-chromatographic method for streamlined AAV manufacturing

Area of Science:

• Biotechnology
• Gene Therapy Manufacturing
• Viral Vector Production

Background:

• Gene therapies are in high demand for treating complex diseases.
• Adeno-associated viruses (AAVs) are key gene delivery vectors but face manufacturing yield challenges (10-30%).
• Current downstream processing relies heavily on chromatography, which can be costly and inefficient.

Purpose of the Study:

• To introduce IsoTag™AAV, a novel purification technology for adeno-associated virus (AAV) production.
• To address the low manufacturing yields and high costs associated with current AAV purification methods.
• To demonstrate the efficacy and cost-effectiveness of IsoTag™AAV compared to traditional techniques.

Main Methods:

• Development of IsoTag™AAV, a proprietary technology utilizing a self-scaffolding recombinant protein reagent.
• Implementation of IsoTag™AAV for purification via scalable filtration processes.
• Head-to-head comparison of IsoTag™AAV with industry-standard affinity chromatography.

Main Results:

• IsoTag™AAV significantly improves AAV manufacturing yields compared to traditional methods.
• The technology enables purification through cost-effective and scalable filtration.
• A 25% reduction in cost of goods was demonstrated for a clinical AAV program using IsoTag™AAV.
• Product quality is maintained with minimal optimization.

Conclusions:

• IsoTag™AAV offers a novel, efficient, and scalable solution for AAV purification.
• This technology represents a significant advancement in overcoming manufacturing bottlenecks for gene therapies.
• IsoTag™AAV has the potential to substantially reduce the cost of goods for clinical AAV programs.