Our Commitment

We are focused on improving the lives of people with inherited retinal diseases through a novel gene therapy approach aimed at stopping disease progression and improving deteriorating vision.

Our patients are at the forefront of everything we do and motivate us to work hard every day, in order to bring important new therapies to the many people who need them.

Toby Stroh

I start my story with some defining comments that were made to me in my 20’s after I’d been misdiagnosed with retinitis pigmentosa. I went to see a consultant and a friend of the family said that I should take my girlfriend, who subsequently became my wife, so she would understand what it entailed and the consultant told me that I was going to go blind by the time I was 50. I remember asking what he meant. He was standing on one side of the room and I was on the other and he said you won’t be able to see me.

To a certain extent this was anticipatory. I was in my early 20s and it’s a long way away, but it still came as a bit of a shock.

The next comment was a couple of years later when I went to see a different consultant as the first one had retired. He sat me down and examined me and told me that I had to stop driving. You move from the anticipatory to the more immediate, because losing the independence of driving brings it rather closer to home with the difficulties present and future.

The third comment came, again in my 20s, in my current firm as a solicitor. I thought I’d let some of my partners know the difficulties I would be facing with my vision at some point in the future. The senior partner said “We will be very sorry to see you go.” This was clearly not what I was hoping to hear.

So, the present is that I can’t drive and the future is that I’m going blind and I’m going to be leaving my job as I’m not going to be able to work.

As mentioned above I was diagnosed incorrectly in my early teens with Retinitis Pigmentosa and I went along for tests every year or every other year to various hospitals and it was awful. I knew my peripheral vision was deteriorating and the results were an extremely acute and unwelcome reminder of what was happening to me. There’s a test called the visual field test, which ascertains how much peripheral vision you’ve got. You sit down looking at a cone and there are pin pricks where the light shines through and it comes in closer to the centre of the cone with the patient being asked to press a button when he or she can see the light being shone through. It’s very depressing not seeing the light. You wait for an increasingly longer time and suddenly you see a light and you click. You come out of that feeling utterly miserable with each year getting worse and wondering why you put yourself through it.

I’d read somewhere that there was some research in retinitis pigmentosa, which I was still under the misapprehension that I had. I went along to see Professor MacLaren who looked into my eyes and informed me that I had choroideremia and invited me to be considered as a candidate for an upcoming clinical trial.

I had the operation on my left eye in February 2012. This was my worse eye, so if things did go disastrously wrong, I’d still have some vision. There was no pain and minimal discomfort and the early results were reported in the Lancet Medical Journal in January 2014.

Jonathan Wyatt

I am lucky. My choroideremia condition did not develop [into a condition] to disadvantage me until I was well into middle age. After initial diagnosis and the news that there was no cure, I ignored the problem until there was no alternative but acceptance.

I had chosen a challenging career as a barrister. In retrospect, others have told me that I achieved some success. On one occasion in court before a difficult, pedantic and vinegary judge, I incorrectly mistook one word for another whilst reading a document. “Can’t you read, Mr. Wyatt,” snapped the man on the bench. I realized it was time to hang up my wig after 29 years of exciting and stimulating court work practice.

The computer beckoned and I enjoyed a new phase of life assisted by the marvels of modern technology.

Having deliberately ignored my eyesight problem [for years,] I now spent time researching it. Professor Seabra was experimenting with the choroid mouse at Imperial College, London, but alas, his concentration was on mice not yet men. My research led me to find that Professor Robert MacLaren of the Oxford University John Radcliffe Hospital, who was contemplating a choroideremia clinical trial. My luck was in again and I became number one Guinea Pig. I was operated on in October 2011 and the BBC has covered some of the outcomes of my treatment.

Wayne Thompson

I was diagnosed with choroideremia when I was 17 years old. At the time, I was told that I had 5-10 years of useful vision left, that I should hand back my driver’s license (a month after passing my test) and that I would not be able to pursue a career in the Royal Air Force. There was very little support offered at the time, and I was left with a very uncertain future.

In my early twenties, I met my now wife, who gave me a very different perspective on my eye condition. Rather than look at the negatives, she looked at the positives and what could be achieved, rather than what couldn’t be. We managed to save up and do some travelling and a lot of other things, which I don’t think I would ever have done if I was left to my own devices.

I suppose I really became aware of the effects of choroideremia in my late twenties. I began to bump into people and objects more frequently, and my night vision was pretty much none existent. My son was born when I was 28, and I was still not sure if I would see him grow up.

In 2003, I was registered blind. One of the benefits of registering was that I had access to mobility training as I had reached the point where I felt that a cane may be necessary in my daily life. It took several more years after that before I finally accepted that a cane was necessary to make life safer for me, and others around me.

Living with choroideremia is sometimes very hard. Being faced on a daily basis with the fact that I was losing my sight and there was nothing I could do about it was very hard. There were some very low days, some frustrating days and some angry days. Being involved in social media was a great help. In the beginning, there was a Yahoo group, and now Facebook. Being in touch with other people, although indirectly, was a great help. To know I wasn’t the only one, and everyone else had the same fears and worries made life a little easier.

In 2008, we travelled to Manchester Royal Eye hospital to see Professor Graeme Black to undergo genetic testing. By this time, we had a daughter, who we knew would be a carrier of the condition, so an exact diagnosis was important for us. The diagnosis was confirmed as choroideremia and I was placed on a register of X chromosome linked conditions and we thought nothing more of it.

In 2011, my wife and I heard the news that clinical trials had started to treat choroideremia. This came out of the blue for us, and it was hard to comprehend at first. Later that year, we travelled to Manchester again to see Professor Black. I was asked if I would like to be assessed for inclusion in the trial. It was not a difficult decision to say yes. This would mean many trips to and from Oxford to see Professor Maclaren and his team. Although these trips were long and tiring, I was fortunate to be accepted onto the trial and received the gene therapy treatment in April 2013. My eye recovered very quickly from the surgery, and I’m hoping the initial results reported in medical journal The Lancet will be sustained.

I am very grateful to Professor MacLaren and his whole team at Oxford. I hope the trial treatment will be a success and that future participants respond well. This trial has given real hope for the first time to sufferers of choroideremia. The real impact of being involved will hit home when the trial becomes a treatment and other men in my situation will benefit knowing that they will not lose their sight. I hope that the success of this trial will also lead to treatments being developed for previously untreatable eye diseases so many more people can benefit.

Disease and Community Resources

We are committed to working together with the academic community and advocacy partners to help bring awareness to our programs and share important information with our patients. We are working closely alongside advocacy groups to gain valuable insights directly from patients as our pipeline of retinal gene therapy progresses.

For more information about the diseases we are aiming to treat and resources for patients, their families and caregivers, please visit the follow pages.

Additional Resources

Links to other websites not operated or controlled by Nightstar are provided for convenience only. By providing these links, we are not endorsing or agreeing with content contained at any of the linked websites

I was diagnosed with choroideremia when I was 17 years old. At the time, I was told that I had 5-10 years of useful vision left, that I should hand back my driver’s license (a month after passing my test) and that I would not be able to pursue a career in the Royal Air Force. There was very little support offered at the time, and I was left with a very uncertain future.

In my early twenties, I met my now wife, who gave me a very different perspective on my eye condition. Rather than look at the negatives, she looked at the positives and what could be achieved, rather than what couldn’t be. We managed to save up and do some travelling and a lot of other things, which I don’t think I would ever have done if I was left to my own devices.

I suppose I really became aware of the effects of choroideremia in my late twenties. I began to bump into people and objects more frequently, and my night vision was pretty much none existent. My son was born when I was 28, and I was still not sure if I would see him grow up.

In 2003, I was registered blind. One of the benefits of registering was that I had access to mobility training as I had reached the point where I felt that a cane may be necessary in my daily life. It took several more years after that before I finally accepted that a cane was necessary to make life safer for me, and others around me.

Living with choroideremia is sometimes very hard. Being faced on a daily basis with the fact that I was losing my sight and there was nothing I could do about it was very hard. There were some very low days, some frustrating days and some angry days. Being involved in social media was a great help. In the beginning, there was a Yahoo group, and now Facebook. Being in touch with other people, although indirectly, was a great help. To know I wasn’t the only one, and everyone else had the same fears and worries made life a little easier.

In 2008, we travelled to Manchester Royal Eye hospital to see Professor Graeme Black to undergo genetic testing. By this time, we had a daughter, who we knew would be a carrier of the condition, so an exact diagnosis was important for us. The diagnosis was confirmed as choroideremia and I was placed on a register of X chromosome linked conditions and we thought nothing more of it.

In 2011, my wife and I heard the news that clinical trials had started to treat choroideremia. This came out of the blue for us, and it was hard to comprehend at first. Later that year, we travelled to Manchester again to see Professor Black. I was asked if I would like to be assessed for inclusion in the trial. It was not a difficult decision to say yes. This would mean many trips to and from Oxford to see Professor Maclaren and his team. Although these trips were long and tiring, I was fortunate to be accepted onto the trial and received the gene therapy treatment in April 2013. My eye recovered very quickly from the surgery, and I’m hoping the initial results reported in medical journal The Lancet will be sustained.

I am very grateful to Professor MacLaren and his whole team at Oxford. I hope the trial treatment will be a success and that future participants respond well. This trial has given real hope for the first time to sufferers of choroideremia. The real impact of being involved will hit home when the trial becomes a treatment and other men in my situation will benefit knowing that they will not lose their sight. I hope that the success of this trial will also lead to treatments being developed for previously untreatable eye diseases so many more people can benefit.

Our Clinical Trials

We are developing a pipeline of novel and potentially curative, one-time retinal gene therapies for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness, and for which there are no currently approved treatments. For more information about our ongoing clinical trials, please visit www.clinicaltrials.gov  and search for NightstaRx. 

If you or someone you know is interested in receiving additional information about our clinical studies, please fill out the form below, and someone from one of our research centers will be in touch.