Üsküdar University takes its gene therapy studies to the international arena…

Synastra Biotechnology, a Türkiye-based biotechnology company established in collaboration with Üsküdar University and Unifon-Biotech Venture Capital Investment Fund (VCF), has signed a significant international collaboration in its research-phase gene therapy program developed for Duchenne Muscular Dystrophy (DMD). Synastra signed an agreement with Switzerland-based biotechnology company NewBiologix for the development of a scalable and reproducible rAAV production infrastructure to be used in the production of the DMD gene therapy candidate. The collaboration represents an important step in bringing gene therapy studies developed within the scientific ecosystem of Üsküdar University and its TRGENMER to the international biotechnology field.

Üsküdar University continues to strengthen its scientific studies in the fields of genetics and biotechnology through international collaborations. Synastra Biotechnology, a Türkiye-based biotechnology company established as a result of the collaboration between Üsküdar University and Unifon-Biotech VCF Venture Capital Investment Fund, has signed an international collaboration in its research-phase AAV-based micro-dystrophin gene therapy program developed for Duchenne Muscular Dystrophy (DMD).

An agreement was reached between Synastra Biotechnology and Switzerland-based NewBiologix, which develops stable cell line technologies for recombinant adeno-associated virus (rAAV) production, for the development of a stable producer cell line for the DMD gene therapy program.

Üsküdar University continues to strengthen its scientific studies in the fields of genetics, biotechnology, and advanced therapy technologies through academic-industrial collaborations and to increase the international visibility of innovative biotechnology projects developed in Türkiye.

Scalable production infrastructure to be developed for DMD gene therapy

Within the scope of the agreement, NewBiologix will establish a stable Research Cell Bank for Synastra's DMD gene therapy candidate and carry out its characterization, using its proprietary Xcell™ Stable Production Platform.

The agreement also includes an option for conversion to a commercial license to support clinical and commercial production in later stages of the program.

With this collaboration, Synastra's expertise in genome engineering, AAV vector design, gene therapy for rare diseases, and translational development aims to be combined with NewBiologix's cell engineering and rAAV production technologies.

Early-stage consideration of production process aimed in gene therapy

By addressing the development of the production process from the early stages of the gene therapy program, the companies aim to establish a genetically defined, reproducible, and scalable production system that can support the transition to clinical application and subsequent commercial supply.

Duchenne Muscular Dystrophy is known as a severe and progressive X-linked neuromuscular disease affecting approximately one in 5,000 male children. The disease results from mutations in the DMD gene, which prevent the production of functional dystrophin protein. This leads to progressive degeneration of skeletal and cardiac muscles.

The production process is critically important in DMD gene therapies. Since systemic treatment in the field of gene therapy may require the use of high quantities of rAAV vectors, rAAV production capacity, production consistency, scalability, and cost are among the decisive factors in the development and widespread accessibility of therapies.

NewBiologix is developing its Xcell™ platform to address these limitations at their source. The platform aims to replace repeated transient transfection methods with genetically engineered stable producer cell lines designed for reproducible and scalable rAAV production.

Synastra continues its work within Üsküdar University's scientific ecosystem

Synastra Biotechnology, which continues its activities with the strategic investment partnership of Üsküdar University and TRGENMER, as well as Unifon-Biotech VCF Venture Capital Investment Fund, develops advanced gene therapies and translational biotechnology programs for rare genetic diseases.

The company's pioneering program consists of a research-phase AAV-based micro-dystrophin gene therapy candidate for DMD. Synastra integrates genome engineering, synthetic biology, rAAV vector design, analytics and CMC strategy, preclinical development, and transition to clinical application.

Synastra, operating within the scientific ecosystem of Üsküdar University's Transgenic Cell Technologies Application and Research Center (TRGENMER), aims to bring gene therapies originating from Türkiye to international production, quality, and regulatory standards.

Gene therapy originating from Türkiye meets international technology

Dr. Cihan Taştan, Vice Chairman of the Board and General Manager of Synastra Biotechnology, stated that the collaboration is important for advancing a gene therapy originating from Türkiye in line with international quality and regulatory standards.

Taştan said, “Synastra was established to transform Türkiye's competencies in genome engineering into gene therapies that can compete internationally for patients with rare genetic diseases. Our program consists of a research-phase AAV-based micro-dystrophin candidate for DMD, and we are establishing the scientific, manufacturing, and translational development processes for this candidate from the outset.”

Taştan stated that they continue their work with the strategic investment partnership of Üsküdar University and TRGENMER, as well as Unifon-Biotech VCF Venture Capital Investment Fund, and added, “The collaboration we have established with NewBiologix is an important step towards advancing a gene therapy originating from Türkiye in line with international quality and regulatory standards. Our ultimate goal is to provide meaningful benefits to patients and their families.”

Dr. Igor Fisch: “Our agreement with Synastra is a significant validation of our goal”

Dr. Igor Fisch, CEO and Co-Founder of NewBiologix, stated that for gene therapy to reach its full potential, production processes must also advance alongside therapy development processes. Fisch said, “Unless production processes evolve with gene therapy, gene therapy will not reach its full potential. DMD, in particular, highlights this challenge because systemic treatment can require very large quantities of rAAV vectors. The traditional transient transfection method is still complex, costly, and difficult to scale consistently. With Xcell™, we integrate the production process from the very beginning of the therapy development process, through stable and genetically defined producer cell lines designed to reduce complexity and variability. This agreement with Synastra is a significant validation of our strategy and our goal to make rAAV production more scalable, reproducible, and economically sustainable.”

Collaboration receives wide coverage in international biotechnology media

The international press release announcing the collaboration between NewBiologix and Synastra was published via GlobeNewswire. The news was distributed to over 250 international industry and media organizations via Roxhill, and the collaboration was also featured in leading biotechnology and pharmaceutical industry publications worldwide. With news published in international outlets such as BioPharm International, The Pharma Letter, Pharmaceutical Executive, DMD Warrior, Swiss Biotech, PharmaBiz, Master’s of Longevity, BioSpace, and Chemocule, Synastra’s work in DMD gene therapy and the collaboration with NewBiologix were brought to the agenda of the global biotechnology ecosystem. This international visibility constituted an important step in increasing the worldwide recognition of Synastra, established in partnership with Üsküdar University, and gene therapy studies developed in Türkiye.

About NewBiologix:

Headquartered in Lausanne, Switzerland, NewBiologix is a biotechnology company focused on making rAAV production industrially viable through its proprietary mammalian cell engineering, genomic, and analytical technologies. The company’s Xcell™ Eng-HEK293 Stable Production Platform incorporates NewBiologix's proprietary Regulatory Network System (RNS). This system is designed to tightly control rAAV production genes during cell proliferation and activate these genes when vector production is required.

Xcell™ is designed to address key limitations of conventional rAAV production, such as consistency, scalability, complexity, and cost, by replacing repeated transient transfection methods with genetically defined stable production systems. NewBiologix's goal is to provide a production platform that can support gene therapy programs from the research phase through clinical development and ultimately commercial production.