The technology enables tissue-specific, precise transgene expression with minimal off-target activity in muscle tissue
Biotechnology company SynGenSys, a specialist in synthetic gene promoter systems, has introduced its Liver.Set library of liver-specific synthetic promoters.
Developed using the company’s informatics and computational design platform, the Liver.Set library comprises a variety of compact, patentable synthetic promoters for liver-targeted gene expression for in vivo gene therapies.
“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,” said SynGenSys CCO Dr Mike Daniels.
Liver.Set enables tissue-specific, precise transgene expression with minimal off-target activity in muscle tissue. This helps to address one of the bottlenecks in biopharma manufacturing and cell and gene therapy development.
Compared to natural liver promoters with fixed activity profiles, sizes and regulatory behaviours, the Liver.Set promoters feature increased activity and design flexibility. The promoters exhibit low levels of activity in HEK293, avoiding viral vector manufacturing issues linked to therapeutic gene expression during packaging. The modular nature of Liver.Set supports customisation and compact sequence lengths, for enhanced compatibility with AAV payloads.
The 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.
