Swiss biotech NewBiologix and Türkiye-based gene therapy company Synastra Biotechnology have announced a new partnership, with NewBiologix developing and producing a stable producer cell line for Synastra's investigational Duchenne muscular dystrophy (DMD) gene therapy programme.
Under the agreement, NewBiologix will leverage its proprietary Xcell Stable Manufacturing Platform to generate and characterise a stable Research Cell Bank for Synastra’s DMD candidate.
In a statement, the duo explained that, by addressing manufacturing early in development, they hoped to establish a genetically defined, reproducible and scalable production system that supports the DMD programme’s progression toward clinical translation and, ultimately, commercial supply.
The pair said that the agreement also contained an option to transition the programme to a commercial license supporting future clinical and commercial manufacturing.
Solving a key issue in DMD gene therapy manufacturing
Duchenne muscular dystrophy is a severe, progressive, X-linked neuromuscular disease affecting approximately one in 5000 male births.
Mutations in the DMD gene prevent production of functional dystrophin, leading to progressive degeneration of skeletal and cardiac muscle.
Manufacturing is essential for DMD gene therapies, as systemic treatments often require high vector doses.
This makes the productivity, consistency, scalability and cost of rAAV critical for developing and ensuring the availability of these therapies.
NewBiologix said it 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, scalable rAAV manufacturing.
Dr Igor Fisch, CEO and co-founder of NewBiologix, 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, said: "Synastra was established to translate Türkiye's capabilities in genomic engineering into internationally competitive gene therapies for patients with rare genetic diseases."
"Our programme 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.
"Our collaboration with NewBiologix 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."
