Developed through a collaborative research effort, two enhancers have now been tested at multiple scales in HEK293 cells using Ascend's EpyQ® two-plasmid AAV Production System and a commercially available 3-plasmid system, with no negative effects on quality parameters such as vg/cap ratio or mispackaged DNA being observed. Enhancer activity was further confirmed using customer plasmids.
- Enhancer 1 has been found to produce up to 3-fold more AAV vector using EpyQ® in Ambr®15 and up to 1.7-fold more for the 3-plasmid system at Ambr®15 scale and in 2L bioreactors.
- Enhancer 2 has been found to produce up to 2.4-fold more AAV using the EpyQ® plasmid system at Ambr®15 scale, and up to 2.1-fold for Ambr®250 scale and 5L bioreactors. This work has now also been replicated in experiments conducted by an external party, interested in licensing.
"Improving AAV productivity without compromising product quality is one of the most persistent challenges in gene therapy manufacturing," said Dr. Markus Hörer, Chief Scientific Officer at Ascend Advanced Therapies. "This enhancer represents a meaningful step forward — not just for our internal platform, but for our partners who depend on scalable and reliable AAV manufacturing. This advancement could allow twice as many patients to be treated per vector batch, reducing cost of goods."
"This advancement reinforces Ascend's position as a science-led CDMO committed to redefining what efficient, high-quality AAV manufacturing can look like," said Dr. Alessandra Rispoli, Chief Executive Officer at Ascend Advanced Therapies. "By integrating this enhancer into our CMC offering, we enable our partners and customers to achieve higher yield, reduced cost per dose, and faster scalability, translating scientific innovation directly into patient and business impact."
Ascend can now integrate the enhancer across its process development and clinical manufacturing workflows, giving their gene therapy developing customers access to more affordable, high quality AAV vectors. The company plans to license the technology to the industry in the coming months.
Data supporting the findings are under patent protection (WO2025087695A1) and builds on work originally published in Molecular Therapy – Methods and Clinical Development in December 2024 (https://www.cell.com/molecular-therapy-family/methods/fulltext/S2329-0501(24)00200-6).
To stay up to date with Ascend, please visit www.ascend-adv.com, follow on LinkedIn, or reach out at business@ascend-adv.com.
About Ascend
Ascend Advanced Therapies (Ascend) is a gene-to-GMP CDMO specializing in high-quality, cost-effective advanced therapy development and manufacturing. Ascend launched in early 2023 with the acquisition of Freeline Therapeutics Munich manufacturing assets and development team with AAV development expertise dating from 1991. In early 2024, Ascend acquired the GMP manufacturing assets from Beacon Therapeutics in Alachua, Florida to build a United States footprint. In late 2024, Ascend and ABL, Inc. aligned to expand development, manufacturing and fill/finish capabilities for gene therapies, oncolytics, vaccines and immunotherapies. The team delivers CMC support backed by decades of therapeutic development experience, helping guide clients from concept to commercialization while balancing yield, quality and cost. Foundational investors include Abingworth, Anjinomoto, Cathay Health, Deerfield, Digitalis Ventures, DCVC Bio, EW Healthcare Partners, 4BIO Capital, Monograph, and Petrichor. Learn more at www.ascend-adv.com.
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