At MeiraGTx, we are pioneering a transformative platform technology that allows precise activation of gene expression with proprietary oral small molecule inducers.
Our inducible gene expression platform is based on synthetic riboswitches rationally designed for mammalian cells, a short RNA hairpin sequence that includes a small molecule binding aptamer that specifically and reversibly binds to the corresponding small molecule inducer.
Our proprietary splicing-based riboswitch platform is superior to other inducible gene expression systems with unprecedented dynamic range, demonstrated ability to regulate any transgene, and allows for the incorporation of any bespoke promoter to achieve desired tissue specificity and expression levels.
In the absence of the inducer, gene expression is off due to the inclusion of a premature stop codon within the spliced message which results in degradation of the message and no protein produced. When the small molecule inducer is present, specific binding of the inducer to the riboswitch aptamer results in splicing out of the entire regulation cassette with the production of stable mRNA and protein expression.
Our rationally designed synthetic riboswitches activate transgene expression via a splicing based expression platform cassette.
Novel synthetic riboswitches are highly dynamic in regulating gene expression in mammalian cells allowing precise activation of gene expression with a small molecule inducer.
Aptamers are interchangeable within the switch and multiple novel synthetic aptamer and novel small molecule pairs have been generated.
Multiple transgenes have been regulated in multiple cell types in vitro and in vivo.
AAV delivered transgene expression is precisely regulated in a dose dependent fashion in vivo via orally available small molecule inducer.
Our riboswitch gene regulation system provides an unprecedented platform for spatial and temporal control of gene therapy with broad implications for the applicability of genetic medicines for treating a wide range of potential disorders not limited to inherited diseases.