MGTX

MeiraGTx Holdings PLC is a vertically integrated, clinical-stage gene therapy company. The Company is focused on three distinct areas of unmet medical need: ocular diseases, including inherited retinal diseases (IRDs), as well as large degenerative ocular diseases, severe forms of xerostomia and neurodegenerative diseases. Its product pipeline includes botaretigene sparoparvovec or bota-vec, for the treatment of X-linked retinitis pigmentosa related to mutations in the retinitis pigmentosa GTPase regulator gene or XLRP-RPGR; AAV-hAQP1 for the treatment of radiation-induced Grade 2/3 Xerostomia; AAV-hAQP1 for the treatment of Sjogren’s Syndrome; AAV-GAD for the treatment of Parkinson’s Disease; AAV-RPE65 for the treatment of RPE65-asociated retinal dystrophy; AAV-CNGB3 and AAV-CNGA3 for the treatment of achromatopsia; AAV-AIPL1 for the treatment of LCA4, and AAV-RDH12 for the treatment of RDH12 mutation-associated retinal dystrophy.