MeiraGTx Provides Clinical Trial Updates for Achromatopsia and Leber's Congenital Amaurosis Ocular G

In addition, MeiraGTx has successfully completed dosing of patients in the second cohort in its clinical trial for Leber's Congenital Amaurosis with RPE65 mutations, also at the Moorfields Eye Hospital in London.

Leber's Congenital Amaurosis (LCA) is a group of autosomal recessive, early-onset retinal dystrophies that cause severe sight impairment in childhood. The underlying deficit in up to 16% of all LCA cases lies in the RPE65 gene, which plays a key role in the regeneration of visual pigment following exposure to light. There is currently no approved treatment for LCA2-RPE65 deficiency.

The timely patient accrual and study initiation in both MeiraGTx inherited retinal disease studies reflects the value of the Company's partnership with Moorfield's Eye Hospital and its access to a world leading patient catchment, as well as the extensive natural history and imaging data gathered on these rare patients.

"We are very excited to be treating inherited blindness by addressing the genetic defect itself. Our aim is to improve the outcome for people with eye diseases that are currently untreatable," said James Bainbridge, MD Ph.D., of Moorfields and UCL.

"MeiraGTx is very pleased to have started a clinical trial in our second genetic ocular program," said Alexandria Forbes, Ph.D., President and CEO of MeiraGTx.  "Our commitment to treating genetic diseases of the eye is a major area of effort for MeiraGTx.  We look forward to continuing our work not only in LCA2 and achromatopsia, but in our other rare eye disease programs as well."

About MeiraGTx 

MeiraGTx is committed to the development of novel gene therapies to transform the lives of patients suffering from acquired and inherited disorders.  The company is developing treatments for ocular diseases, including rare inherited blindness and age-related macular degeneration (AMD).  MeiraGTx is also establishing treatments for xerostomia, a frequent and debilitating side effect of radiation treatment used in head and neck cancers, as well as certain neurodegenerative diseases.  In addition, MeiraGTx is developing novel gene regulation platforms that promise to transform the way gene therapy can be applied and create new paradigms for biologic therapeutics.

About Moorfields Eye Hospital

Moorfields Eye Hospital NHS Foundation Trust is the leading provider of eye health services in the UK and a world-class center of excellence for ophthalmic research and education. We have a reputation, developed over two centuries, for providing the highest quality of ophthalmic care. Our 1,800 staff are committed to sustaining and building on our pioneering legacy and ensuring we remain at the cutting edge of developments in ophthalmology.

About UCL

The UCL Institute of Ophthalmology conducts cutting-edge science, attracting research workers of the highest international caliber. The most recent national Research Excellence Framework has once more confirmed that they have the strongest Clinical Medicine research of any British university. Independent auditors have shown that their partnership with Moorfield's Eye Hospital is the most productive in the world.