NewBiologix and Synastra Partner to Advance Stable rAAV Manufacturing for Duchenne Muscular Dystrophy Gene Therapy
核心洞察
NewBiologix (搜索) and Synastra Biotechnology (搜索) have agreed to develop a stable producer cell line for Synastra's investigational DMD gene therapy candidate using the Xcell Stable Manufacturing Platform.
The collaboration aims to establish a genetically defined, reproducible, and scalable rAAV production system to support clinical translation and eventual commercial supply.
DMD is a severe X-linked neuromuscular disease affecting roughly one in 5,000 male births, and systemic gene therapy may require some of the highest vector doses in the field.
NewBiologix (搜索) S.A., a Swiss biotechnology company pioneering stable cell line technologies for recombinant adeno-associated virus (rAAV) manufacturing, has announced an agreement with Synastra Biotechnology (搜索) Inc. to develop a stable producer cell line for Synastra's investigational Duchenne muscular dystrophy (搜索) (DMD) gene therapy program. Under the agreement, NewBiologix will use its proprietary Xcell Stable Manufacturing Platform to generate and characterize a stable Research Cell Bank for Synastra's DMD gene therapy candidate, with 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 (搜索)'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 toward clinical translation and, ultimately, commercial supply.
Addressing a Critical Manufacturing Challenge in DMD Gene Therapy
Duchenne muscular dystrophy (搜索) 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 (搜索), leading to progressive degeneration of skeletal and cardiac muscle.
Manufacturing is particularly critical for DMD 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 (搜索) 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.
Leadership Perspectives on the Partnership
Dr. Igor Fisch, CEO and Co-Founder of NewBiologix (搜索), said: "Gene therapy will not reach its full potential unless manufacturing evolves with it. DMD makes this challenge particularly clear because systemic treatment can require very 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."
Dr. Cihan Taştan, Deputy Chairman of the Board and General Manager of Synastra Biotechnology (搜索), said: "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 (搜索) 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. Our collaboration with NewBiologix (搜索) is an important step toward advancing a gene therapy originating from Türkiye in line with international quality and regulatory standards, with the ultimate goal of delivering meaningful benefits to patients and their families."
About the Companies
Synastra Biotechnology (搜索) Inc. is a Türkiye-based biotechnology company established through a collaboration between Üsküdar University and Unifon-Biotech GSYF Venture Capital Investment Fund. Led by molecular biologist and genetic engineer Dr. Cihan Taştan, Synastra develops advanced gene therapies and translational biotechnology programs for rare genetic diseases. Its lead program is an investigational AAV-based micro-dystrophin (搜索) gene therapy candidate for DMD, integrating genomic engineering, synthetic biology, rAAV vector design, analytical and CMC strategy, preclinical development, and clinical translation.
NewBiologix (搜索) S.A., headquartered in Lausanne, Switzerland, focuses on industrializing rAAV manufacturing through proprietary mammalian cell engineering, genomics, and analytics. Its Xcell Eng-HEK293 Stable Manufacturing Platform incorporates NewBiologix's proprietary Regulatory Network System (RNS), engineered to tightly control rAAV production genes during cell expansion and activate them when vector production is required. By replacing repeated transient transfection with genetically defined stable production systems, Xcell is designed to address key limitations of conventional rAAV manufacturing, including consistency, scalability, complexity, and cost.