NewBiologix and Synastra Partner on Stable AAV Manufacturing for Duchenne Gene Therapy

Switzerland-based NewBiologix and Türkiye-based Synastra Biotechnology have entered into an agreement to develop a stable producer cell line for Synastra's investigational Duchenne muscular dystrophy (DMD) gene therapy candidate, using NewBiologix's proprietary Xcell stable manufacturing platform, the company announced September 2, 2026.¹ The agreement also includes an option to transition the program to a commercial license supporting future clinical and commercial manufacturing.

"Gene therapy will not reach its full potential unless manufacturing evolves with it," said Igor Fisch, PhD, CEO and co-founder of NewBiologix, in a company press release.¹ "DMD makes this challenge particularly clear because systemic treatment can require very large quantities of rAAV [recombinant adeno-associated virus] vector. Conventional transient transfection remains complex, costly and difficult to scale consistently."

Why is manufacturing especially challenging for DMD gene therapy?

DMD is a severe, progressive, X-linked neuromuscular disease caused by mutations in the DMD gene that prevent production of functional dystrophin, leading to degeneration of skeletal and cardiac muscle. The disease affects an estimated 1 in 5000 male births, consistent with pooled global birth-prevalence estimates from peer-reviewed epidemiological research.¹^,² Because systemic DMD gene therapy can require some of the highest vector doses in the field, rAAV productivity, consistency, scalability, and cost are decisive factors in developing and broadly delivering these therapies, according to NewBiologix.¹

Manufacturing complexity tied to high-dose AAV delivery is a challenge across the gene therapy field, not just in DMD, industry experts note. "If you can bring down the dose of an AAV by 50-fold, it would really help alleviate some of the toxicity challenges and probably also make manufacturing simpler," said Erik Wiklund, PhD, CEO of Circio, in an earlier interview with BioPharm International® where he discussed a different approach to reducing AAV dose burden.³

How does the Xcell platform work?

NewBiologix's Xcell platform is designed to replace repeated transient transfection with genetically engineered, stable producer cell lines intended to enable more reproducible and scalable rAAV manufacturing. The platform incorporates the company's proprietary Regulatory Network System, which is engineered to tightly control rAAV production genes during cell expansion and activate them only when vector production is required.¹ Under the agreement, NewBiologix will generate and characterize a stable research cell bank for Synastra's DMD candidate.¹ NewBiologix focuses on industrializing rAAV manufacturing through proprietary mammalian cell engineering, genomics, and analytics.¹

What is Synastra's DMD program?

"Synastra was established to translate Türkiye's capabilities in genomic engineering into internationally competitive gene therapies for patients with rare genetic diseases," said Cihan Taştan, PhD, deputy chairman of the board and general manager of Synastra Biotechnology, in the release.¹ "Our program is an investigational AAV-based micro-dystrophin candidate for DMD, and we are building its scientific, manufacturing, and translational pathway from the outset. Together with Üsküdar University, including TRGENMER, and the strategic investment partnership of Unifon-Biotech GSYF Venture Capital Investment Fund, we are connecting construct design and preclinical development with scalable rAAV manufacturing." The company aims to build a broader pipeline of AAV-based genetic medicines for rare diseases beyond its lead DMD program.

What are the limitations?

This agreement covers early-stage cell line development rather than clinical manufacturing. The companies did not disclose a clinical trial timeline, dosing strategy, or regulatory pathway details for Synastra's DMD candidate. In addition, the commercial license option is not guaranteed to be exercised.

References

1. NewBiologix. NewBiologix and Synastra Biotechnology partner to advance stable rAAV manufacturing for Duchenne muscular dystrophy gene therapy. Press release. Published September 2, 2026. Accessed September 2, 2026. https://www.newbiologix.com/news/newbiologix-and-synastra-biotechnology-partner-to-advance-stable-raav-manufacturing-for-duchenne-muscular-dystrophy-gene-therapy
2. Crisafulli S, Sultana J, Fontana A, Salvo F, Messina S, Trifirò G. Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis. Orphanet J Rare Dis. 2020;15(1):141. doi:10.1186/s13023-020-01430-8
3. Schoenthaler E, Wiklund E. Circular RNA may transform AAV gene therapy, says Circio CEO Erik Wiklund. BioPharm International. Published May 19, 2026. Accessed September 2, 2026. https://www.biopharminternational.com/view/circular-rna-may-transform-aav-gene-therapy-says-circio-ceo-erik-wiklund