Control of gene expression is fundamentally important in the development of both in vivo gene therapy and ex vivo gene modified cell therapy products. Synpromics' technology also enables the ability to control gene expression through inducible systems. Regulation at the tissue level through various inducers creates the unique opportunity for therapeutic developers to improve product function through targeted expression level and cell type(s) selectivity.
- Tissue specific gene expression control
- Inducible promoters: tight control and tuneable activity levels
- Repressible promoter: turning off constitutive genes
- In vivo application using any gene therapy vector
- Inducers and repressors: FDA approved drugs with strong safety record
- Screening platform developed
- Can screen against inducer(s) of choice
Synpromics has developed new technologies that enable the creation of tissue specific inducible and repressible systems. These systems are comprised of a single component and therefore offer an advantage over commonly used 2 component systems, such as Tet on/off, as they can easily be incorporated into a single AAV virus. The systems are tightly regulated, tuneable and have a large dynamic range, showing expression strengths of many times commonly used viral promoters. In addition, gene expression is controlled by FDA approved small molecules which are safe for human use. The small size, dynamic range, tuneability and tightness of control makes Synpromic’s tissue specific inducible and repressible systems ideal for use in gene therapy.
Synpromics' Technology Improves Gene Therapeutics
The technology created at Synpromics enables the design of novel synthetic promoters:
- selective to tissue, single, dual or multiple cell types, developmental stage, or cell state
- variable expression levels - strong, medium, low activity
- various tailored sizes (bp)
- designed to be regulatable, inducible, or have a safety switch feature
Synpromics' novel approach to synthetic promoter development means that it is now possible to develop new promoters to improve therapeutic cell and gene therapy products. We can either select and out-license candidates from existing promoter libraries or alternatively engage in a design and development program to deliver promoters with particular criteria that are unique to the specific application.