MGTX

MeiraGTx Holdings plc is a clinical-stage therapy company. The Company develops gene therapy treatments for a range of inherited and acquired disorders. It is focused on developing therapies for ocular diseases, including rare inherited blindness, as well as Xerostomia, radiation treatment for head and neck cancers and neurodegenerative diseases, such as amyothrophic lateral sclerosis (ALS). It is also developing transformative technology to enable the use of small molecules to turn gene therapy product candidates on and off. It is focused on three areas of unmet medical needs, including inherited retinal diseases, severe forms of xerostomia and neurodegenerative diseases. Its product candidates include AAV-CNGB3, AAV-CNGA3, AAV-RPGR, AAV-RPE65, A006, AAV-AQP1 and AAV-UPF1.