Rockville, Maryland — January 8, 2026 — AAVnerGene Inc. (AAVnerGene) today announced the launch of AAVone®2.0, the next-generation evolution of its proprietary AAVone® single-plasmid AAV production system, delivering significant advances in vector quality, manufacturing efficiency, and cost effectiveness.
AAVone®2.0 achieved ~1×1016 genome copies (GC)/L of cell culture and greater than 70% full capsids at harvest, representing a step-change improvement over AAVone®1.0 and conventional multi-plasmid AAV production systems that typically yield a high proportion of empty capsids.
From High Productivity to High-Quality Vector Output
The first-generation AAVone®1.0 platform established AAVnerGene as a leader in upstream AAV productivity, achieving up to ~7×1015 GC/L in HEK293-based suspension systems while simplifying production through a single-plasmid architecture.
Rongze Yang et al., AAVone: A Cost-Effective, Single-Plasmid Solution for Efficient AAV Production with Reduced DNA Impurities. Molecular Therapy Nucleic Acids Volume 36, Issue 2, 10 June 2025
AAVone®2.0 builds on this foundation by significantly improving genome encapsidation efficiency, thereby delivering:
- >70% full capsid ratios at harvest
- 30-400% additional titer increases, depending on serotype
- Improved dose efficiency and downstream manufacturability
These improvements translate directly into lower plasmid demand, reduced purification burden, fewer processing steps, and meaningfully lower cost of goods, improving the clinical and commercial viability of AAV gene therapy programs.
Evidence of Improved Vector Quality
Vector quality improvements with AAVone®2.0 are supported by both qualitative visualization and quantitative single-particle analysis in HEK293-based production systems.
Using AAV9 as an example:
- AAVone®1.0 produced ~7×1015 GC/L with ~35% full capsids
- AAVone®2.0 increased productivity by ~30% and raised the full-capsid ratio to ~70%, representing an approximate two-fold increase in functional vector content within the same production system
These gains enable production of clinical-scale quantities of high-quality AAV using smaller culture volumes and simplified downstream purification, significantly reducing plasmid, consumable, and labor costs.
Broad Serotype Performance and Platform Robustness
Beyond AAV9, AAVone®2.0 demonstrates broad applicability across multiple serotypes, including historically challenging ones.
Internal validation studies with our HEK293one cells show that AAVone®2.0 improves total vector yield across several serotypes, with up to four-fold productivity gains for AAV5, while maintaining strong performance with AAV9 (use PEIone as transfection reagent). Additional improvements have also been observed in other HEK293-based cell lines, including VPC2.0 (data on file).
Qizhao Wang, Ph.D., Chief Technology Officer of AAVnerGene, noted: “We are encouraged by the recent progress with AAVone®. Through systemic optimization of vector designs, cell lines, culture media, and transfection reagents and enhancers, we have developed a highly efficient and cost-effective AAV manufacturing platform. AAVone®2.0 delivers industry-leading productivity alongside markedly improved full-particle ratios, while remaining fully compatible with existing GMP manufacturing workflows. As a result, the platform can be readily adopted by gene therapy developers seeking to improve vector quality, scalability, and manufacturing economics.“
Availability and Partnership Opportunities
AAVone®2.0 is currently available for research-use evaluation.
AAVnerGene engages partners seeking to improve dose efficiency, vector quality, and manufacturing robustness at the platform level, rather than through incremental process optimization. Partnership models include program-specific licensing and CDMO technology transfer, aligned with the partner’s existing development and manufacturing strategies.
“AAV gene therapy continues to face significant commercialization challenges, with manufacturing cost and vector quality among the most critical constraints,” said Daozhan Yu, PhD, Chief Executive Officer of AAVnerGene. “Addressing these limitations has been a core objective for AAVnerGene since our founding. With AAVone®2.0, we believe we have reached an important milestone – delivering materially improved full-particle ratios and manufacturing efficiency on a platform ready for broader adoption. AAVone®2.0 is now available for program-specific licensing and CDMO technology transfer, and we look forward to engaging with partners who share our focus on scalable, clinically viable AAV development.”
About AAVnerGene Inc.
AAVnerGene is a Maryland-based biotech company focused on innovative solutions to address bottlenecks in AAV gene therapy, including manufacturing, toxicity, qualification, and delivery. Its proprietary solutions—including AAVone®, the AAV-Q (potency and rcAAV assay platform), and the ATHENA capsid engineering platform—are designed to improve productivity, quality, scalability, efficiency, and safety in AAV gene therapy drug development.
Media & Business Inquiries:
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