#USA #Minneapolis #gene therapy #Ascend Advanced #AAV Production

Ascend Unveils Innovative Enhancers to Boost AAV Yields

Ascend Advanced Therapies has made headlines with its groundbreaking data showcasing the effectiveness of two proprietary enhancers aimed at improving adeno-associated virus (AAV) yields. This remarkable development signals a leap forward for the manufacturing processes associated with gene therapy. The ability to increase productive yields without compromising quality marks a major milestone for the biotechnology industry.

The enhancers were developed through extensive collaborative research and have been tested across multiple scales using HEK293 cells with Ascend’s innovative EpyQ® two-plasmid AAV production system, as well as a widely used three-plasmid system. Remarkably, the introduction of these enhancers did not adversely affect crucial quality parameters such as the vector genome to capsid ratio or the occurrence of mispackaged DNA.

Enhancer Performance

Data reveals that Enhancer 1 can yield up to three-fold more AAV vector using the EpyQ® system within the Ambr®15 scale, and 1.7-fold more for the three-plasmid system at the same scale and in 2L bioreactors. On the other hand, Enhancer 2 has shown to enhance AAV yields by up to 2.4-fold when using the EpyQ® system at Ambr®15 scale, and 2.1-fold in larger Ambr®250 scales and 5L bioreactors. These improvements have also been validated through experiments conducted by an external party interested in potentially licensing this technology.

Dr. Markus Hörer, Ascend’s Chief Scientific Officer, commented on the significant challenge of enhancing AAV productivity in gene therapy manufacturing. He noted, “This enhancer represents a meaningful step forward for our internal platform and our partners relying on scalable AAV manufacturing. It enables a potential doubling in patient treatments per vector batch, thereby reducing the associated costs.”

Dr. Alessandra Rispoli, Ascend’s Chief Executive Officer, further emphasized the strategic importance of these advancements: “Our commitment as a science-led contract development and manufacturing organization (CDMO) is to redefine efficient and high-quality AAV manufacturing standards. By integrating this enhancer into our offerings, we empower our partners to achieve greater yields, lower costs per dose, and enhance scalability, translating scientific innovation into real-world impacts for patients and businesses.”

Ascend's ability to implement these enhancers within its process development and clinical manufacturing workflows grants its gene therapy clients access to high-quality AAV vectors at a more affordable rate. In anticipation of future relationships, the company plans to license this innovative technology to the industry within the upcoming months.

The supporting data for this research is currently protected under patent (WO2025087695A1) and builds upon previously published work in the journal Molecular Therapy – Methods and Clinical Development from December 2024. Interested parties can learn more about Ascend by visiting Ascend's website or following the company on LinkedIn for updates on their advancements.

Ascend Advanced Therapies specializes in gene-to-GMP contract development and manufacturing. Since its establishment in early 2023, the company focuses on the cost-effective and high-quality development and manufacturing of advanced therapies. Their strategic acquisitions in relevant facilities and expertise enhance their capabilities, aiming to support clients through the journey from concept to commercialization while keeping a balance in yield, quality, and overall cost.

With significant backing from notable investors and a resolute commitment to innovation, Ascend Advanced Therapies is poised to make a lasting impact on the gene therapy landscape, paving the way for enhanced patient outcomes and efficient manufacturing processes across the industry.