Dynamic range: the key to MeiraGTx’s quest to control genes with pills 

As MeiraGTx assembles its suite of technologies to control gene expression with oral pills, it has homed in on dynamic range as the element that will allow it to succeed where others have failed. Having built tailored, small molecule-activated riboswitches into its gene therapies, the company says it has achieved lower baselines and higher maximum expression than past attempts at gene control, opening the door to precision dosing of any biologic in vivo.

This preclinical platform is the last pillar of optimization in the clinical-stage company’s toolbox, according to CEO Alexandria (Zandy) Forbes. “There’s one major technology that has never been achieved, and that is to control gene expression with an oral pill,” she told BioCentury. ...