Switzerland-headquartered NewBiologix signed an agreement with Synastra Biotechnology to develop a stable producer cell line for Synastra’s investigational Duchenne muscular dystrophy (DMD) gene therapy program. Synastra, based in Turkey, was established through a collaboration between Üsküdar University and Unifon-Biotech GSYF Venture Capital Investment Fund.
NewBiologix will use its Xcell™ stable manufacturing platform to generate and characterize a research cell bank for Synastra’s DMD gene therapy candidate. The agreement also provides an option to transition the program to a commercial license supporting future clinical and commercial manufacturing.
The collaboration brings together Synastra’s expertise in genomic engineering, AAV vector design, rare-disease gene therapy, and translational development with NewBiologix’s proprietary cell engineering and rAAV manufacturing technologies. By addressing manufacturing early in development, the companies aim to establish a genetically defined, reproducible, and scalable production system capable of supporting the program’s progression towards clinical translation and, ultimately, commercial supply.
Critical manufacturing challenge
Duchenne muscular dystrophy is a severe, progressive, X-linked neuromuscular disease affecting approximately one in 5,000 male births. It is caused by mutations in the DMD gene that prevent the production of functional dystrophin, leading to progressive degeneration of skeletal and cardiac muscle.
Manufacturing is particularly critical for DMD and, in fact, all gene therapies. Systemic treatment may require some of the highest vector doses in the field, making rAAV productivity, consistency, scalability and cost decisive factors in the development and broad availability of these therapies.
NewBiologix is developing Xcell to address these constraints at their source by replacing repeated transient transfection with genetically engineered, stable producer cell lines designed for reproducible and scalable rAAV manufacturing, according to Igor Fisch, PhD, CEO and co-founder of NewBiologix.
“Gene therapy will not reach its full potential unless manufacturing evolves with it,” he says. “DMD makes this challenge particularly clear because systemic treatment can require large quantities of rAAV vector. Conventional transient transfection remains complex, costly and difficult to scale consistently.
“With Xcell, we integrate manufacturing into therapy development from the outset, through stable, genetically defined producer cell lines designed to reduce complexity and variability. This agreement with Synastra is an important validation of our strategy and of our ambition to make rAAV manufacturing more scalable, reproducible, and economically sustainable.”
“Synastra was established to translate Türkiye’s capabilities in genomic engineering into internationally competitive gene therapies for patients with rare genetic diseases. 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,” adds Cihan Tastan, PhD, deputy chairman of the board and general manager of Synastra. “We are connecting construct design and preclinical development with scalable rAAV manufacturing.”
