3 March 2020
• PRIME designation granted based on clinical data from ongoing Phase 1/2 trial of AAV-RPGR
• AAV-RPGR is the only XLRP treatment in development to be awarded PRIME designation
UCL spinout MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, has announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Advanced Therapy Medicinal Product (ATMP) designations to AAV-RPGR, MeiraGTx’s investigational gene therapy for the treatment of x-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.
MeiraGTx (Nasdaq: MGTX) is a vertically integrated, clinical stage gene therapy company with six programs in clinical development and a broad pipeline of preclinical and research programs. MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team, MeiraGTx has taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTx’s initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, neurodegenerative diseases and severe forms of xerostomia. Though initially focusing on the eye, central nervous system and salivary gland, MeiraGTx intends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases.
For more information, please visit www.meiragtx.com