Validated, tissue-specific library enables customisable and controllable liver-targeted gene expression with high specificity and minimal off-target activity in muscle tissue
SynGenSys, a biotechnology company designing synthetic gene promoter systems to address critical bottlenecks in biopharma manufacturing and enhance cell and gene therapy development, today announced the launch of its Liver.SET™ library of liver-specific synthetic promoters. Developed using SyngenSys’ proprietary informatics and computational design platform, the Liver.SET library comprises a range of compact, patentable synthetic promoters for liver-targeted gene expression for in vivo gene therapies.
Liver.SET overcomes key challenges in cell and gene therapy development by enabling tissue-specific, precise transgene expression with minimal off-target activity in muscle tissue. Validated both in vitro and in vivo for target specificity, the promoters display a marked increase in activity and design flexibility in comparison to natural liver promoters with fixed activity profiles, sizes, and regulatory behaviours. The promoters exhibit low levels of activity in HEK293, avoiding viral vector manufacturing issues linked to therapeutic gene expression during packaging, while the modular architecture of the promoters further supports rapid customisation alongside compact sequence lengths yielding enhanced compatibility with AAV payloads.
Following the launch of SynGenSys’ first tissue-type specific library, NK.SET1, which supports the development of natural killer cell therapies, Liver.SET further demonstrates the ability to design tissue-specific promoters with customisable designs for therapeutic applications. The demonstrated specificity of SynGenSys’ promoters provides a foundation for the development of promoter libraries designed to target and de-target tissues including muscle, retina and CNS. The commercialisation of the validated libraries also demonstrates SynGenSys’ capabilities for tailored promoter design for targeted therapeutics, facilitating the development of safer, more effective cell and gene therapies.
Dr Mike Daniels, Chief Commercial Officer, SynGenSys, commented: “The launch of Liver.SET™ represents another significant milestone for SynGenSys, demonstrating that our platform can deliver synthetic promoter solutions for real gene therapy development needs. We see this as a key enabler for in vivo gene therapies, and a reliable, validated starting point for deeper collaboration with developers seeking to design novel therapeutics with enhanced precision and safety.”
For further information please visit: https://tinyurl.com/liverSET
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