Gene therapy
Target your clarification step to upgrade your process
Adeno-associated viruses (AAV) are a leading platform for the delivery of gene therapies.
However, the use of endonuclease treatment of AAV lysate for clarification can cost as much as $100,000 USD per 500-L batch.1,2 Eliminating the endonuclease step while maintaining quality and yield could improve process efficiency and reduce the cost of clarification. Process development may also be able to go from discovery to production more quickly by employing a streamlined approach with reliable solutions in sizes that match each development stage.
As you strive to bring accessible treatments to fruition, consider how an innovative clarification solution can deliver the DNA reduction and AAV recovery you need.
References
1 Garima Thakur, Sheldon Mink, Hanne Bak, and Andrew D. Tustian, “Improving process efficiency to reduce cost-of-goods per dose in manufacturing of recombinant AAVs,” Cell & Gene Therapy Insights, 10, no.3 (2024): 479-499, DOI: 10.18609/cgti.2024.061. https://www.insights.bio/cell-and-gene-therapy-insights/journal/article/3192/Improving-process-efficiency-to-reduce-cost-of-goods-per-dose-in-manufacturing-of-recombinant-AAVs
2 Garima Thakur, Sheldon Mink, Hanne Bak, and Andrew D. Tustian, “Single-use chromatographic clarification to eliminate endonuclease treatment in production of recombinant adeno-associated viral vectors,” Separation and Purification Technology, 2024, 128557, DOI: 10.1016/j.seppur.2024.128557. Single-use chromatographic clarification to eliminate endonuclease treatment in production of recombinant adeno-associated viral vectors - ScienceDirect
Complimentary webinar: Emerging trends and challenges in cell and gene therapy
Innovations in cell and gene therapy have the potential to treat, and even cure, serious diseases that have few safe and effective treatment alternatives. These specialized medicines work with our own bodily systems and activate them to fight diseases, including cancers, central nervous system disorders and musculoskeletal conditions. Yet, there are hurdles to clear in both discovery and production processes, which, once overcome, will facilitate bringing these life-altering medicines to the patients who need them most. Key to meeting these challenges will be understanding the trends that exist in this exciting field.
View our complimentary, on-demand webinar presented by the School of Purification, “Emerging Trends and Challenges in Cell and Gene Therapy,” to gain insight on these trends, and learn how to gain efficiencies while overcoming development and manufacturing challenges. An important part of the program is a case study on how chromatographic clarifiers can provide solutions for gene therapy processes.
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