MeiraGTx: restoring sight and hope through pioneering gene therapies

Problem to be solved

Inherited retinal diseases are caused by hundreds of different genetic mutations and collectively affect millions of people worldwide. For many conditions, there have historically been no approved treatments, and patients can face progressive sight loss leading to legal blindness in early adulthood. 

There is a significant unmet need for therapies that address the underlying genetic cause and preserve, restore or stabilise vision.

Solution

MeiraGTx is a genetic medicines company developing gene therapies across ophthalmology and other disease areas, with several programmes rooted in research from UCL’s Institute of Ophthalmology. Its ophthalmology pipeline includes treatments for inherited retinal diseases such as X-linked retinitis pigmentosa (XLRP), as well as early-stage programmes including Leber Congenital Amaurosis-4 (LCA4), where delivering a healthy copy of the affected gene aims to restore the function of light-sensitive retinal cells.

How UCL Ventures helped

MeiraGTx’s relationship with UCL built on earlier translational work and spinout activity connected to ocular gene therapy research. UCL Ventures supported the commercial pathway by helping structure collaborations and licensing arrangements and by maintaining links between the company and the UCL clinical and research community. The UCL Technology Fund (UCLTF) also participated in funding rounds that helped progress clinical programmes and build the capabilities needed for development and manufacturing. 

Michel Michaelides is the trial investigator and Professor of Ophthalmology at the University College London. He presented data to the American Academy of Ophthalmology 2020 conference, showing that the treatment was well-tolerated and demonstrated sustained or increased vision improvement in patients with XLRP: “The fact that we’re actually seeing improvements in vision is very unexpected in many ways, but clearly game-changing.”

Where is MeiraGTx now?

MeiraGTx announced in 2024 that Khadijah Chaudhry, 3, from Hull, was the first patient at Evelina London to have this therapy. Since the trial, four more young children have gained life-changing improvements in sight through UCL Institute of Ophthalmology and Moorfields Eye Hospital, with the support of MeiraGTx. 

Professor Michel Michaelides, UCL Institute of Ophthalmology, and Consultant Retinal Specialist at Moorfields Eye Hospital, said: “We have, for the first time, an effective treatment for the most severe form of childhood blindness, and a potential paradigm shift to treatment at the earliest stages of the disease. The outcomes for these children are hugely impressive and show the power of gene therapy to change lives.” 

The parents of one of the children, Jace (pictured) said that after the operation, Jace was immediately spinning, dancing and making the nurses laugh. “He started to respond to the TV and phone within a few weeks of surgery and, within six months, could recognise and name his favourite cars from several metres away; it took his brain time, though, to process what he could now see. Sleep can be difficult for children with sight loss, but he falls asleep much more easily now, making bedtimes an enjoyable experience.” 

Another MeiraGTx programme is a gene therapy treatment for X-linked retinitis pigmentosa (XLRP), an incurable genetic disease that causes blindness in men and affects approximately one in 15,000 people. Meira’s therapeutic bota-vec (botaretigene sparoparvovec) is currently in phase 3 clinical trials (Lumeos XLRP study). 

This product was acquired by J&J innovative Medicine (formerly Janssen Pharmaceuticals) in December 2023.