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Gene Therapies for Stargardt Disease and Wet AMD Deemed Safe Thus Far

Researcher looking through microscopeIf you’re going on a cross-country road trip, the highlight of your journey is not the safety check. Putting air in the tires, topping off the brake and power-steering fluids, making sure the front and back lights are working properly and adjusting the mirrors are not cause for raucous celebration.

But without taking these safety precautions, your trip could turn out very badly; you not only might not get where you’re going, you might get injured or worse. Taking a “safety first” approach is important in many of life’s endeavors, including the development of sight-saving treatments for retinal diseases.

That’s why I was pleased to recently learn that Oxford BioMedica’s StarGen™ gene therapy for Stargardt disease and RetinoStat® gene therapy for wet age-related macular degeneration received positive interim safety reviews from a Data and Safety Monitoring Board, an independent panel of science and medical experts, which monitors patient safety and treatment efficacy data for a clinical trial. The Foundation requires a DSMB for all its clinical trials, to ensure that no study participant is unnecessarily put in harm’s way.

After 12 months, eight patients in the Phase I/IIa StarGen clinical trial receiving the lowest dose (dose level 1) treatment have experienced no adverse events, and the DSMB supports treating four additional patients with a higher dose (dose level 2). If the safety profile of StarGen continues to be good, four additional patients will receive the highest dose (dose level 3).

In the Phase I RetinoStat clinical trial, a total of nine patients have been treated thus far with no adverse events. Three patients have been treated for each of three different dosing levels, with the first patients having received the dose level 1 treatment 18 months ago. In addition, the treatments are expressing the therapeutic proteins they were designed to express. The DSMB supports the treatment of nine additional patients in the RetinoStat trial.

The DSMB has not issued a safety review yet for the Phase I/II clinical trial of UshStat®, Oxford BioMedica’s gene therapy for Usher syndrome type 1B. That trial began in March 2012, so I wouldn’t expect a report until next year.

I know for those of you losing vision to retinal diseases, an announcement of safety for a treatment may not be the big news that you hope for, and that is perfectly understandable. You want your vision saved or restored. But please keep in mind that safety is the primary goal of these early-stage studies, and without safety, you have nothing.

Gene therapies, with as much promise as they hold, are still cutting-edge treatment approaches – which is why you can’t currently walk into a doctor’s office and ask for one. But our goal at the Foundation Fighting Blindness is to develop it to the point where your doctor says, “Gene therapy for your vision — no problem.” Safety is an essential step toward getting there.

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33 Responses to 'Gene Therapies for Stargardt Disease and Wet AMD Deemed Safe Thus Far'

  1. Eric says:

    Thank you Dr. Rose! I am closely following your work, that of ACT, and gene therapy trials. My wife suffers from Stargart’s and we are hoping to have children soon. I pray that my children won’t be affected (since I am a carrier of a mutation in my ABCA4 gene), and that my wife’s vision is one day restored!

    • EyeOnTheCure says:

      Thanks for your support Eric. As you said, it can be difficult waiting for the cures and treatments that are so badly needed. During this time, we wish you and your family all the best.

  2. vickie hook says:

    my granddaughter hsa stargardt disase. kara was 18 in her last of school when the disase hit her. she wii be 21 next. as a grandmother l hope and pray there is cuer for kara and other like her. befor gandpa and i psss away.

    • EyeOnTheCure says:


      Thanks for sharing your story with us. Like you, we are hoping for a cure each day and doing all we can to make it happen. We wish you and your family all the best during this difficult time.

  3. vickie hook says:

    pleses send me anything on stargardt disease. our we getting close for hope? thanks

    • EyeOnTheCure says:

      Vickie, please visit the Stargardt section of our website here: Please be sure to visit the column towards the left of this page. There you will see a link titled ‘Research articles.’ Please click that. The articles on the next page will provide information on the most recent work that is being done to fight Stargardts. We hope this information is helpful.

  4. Mark says:

    Thanks for your dedication to finding a cure for these retinal diseases DR. Rose! Keep up the good work we our depending on you and others to find a cure for these types of diseases. I will do my part in helping to raise money & donating to the foundation because that is my daughter that Vickie is talking about in the previous mesages. It has really been tough on my daughter she is currently trying to get a degree in Vetinary Technology which has been a dream of hers to help animals, so please do what you can to help her and others fulfill their dreams.
    Thank you very much

    • EyeOnTheCure says:

      Thanks for your kind words and support Mark. We and the researchers in the field certainly cannot do it without you and so we are so grateful for your passion and dedication. We wish you, your daughter, and the rest of your family, all the best!

  5. tharindu ganganath says:

    I am also a stargardt diesease patient live in sri i am 23 years old. i am also hoping for this stem cell therophy. i think it will help our disease one now we have a hope our low vision. these clinical trials will treat us one day…….
    i like to share other stargardt patients” status and we can get together
    contact me

  6. Jon Power says:

    Thank you for the update and this is very positive. I am 33 and will keep close watch on your work with Stargadts. Thank you so much

  7. samruddhi says:

    i just wana know whether there is any hope for d treatment of stargardts disease……will i be able to see properly someday??? any treatment????

  8. Kadher says:

    Hi ,
    I have stargardts and i was just wondering … mentioned this is cutting edge technology and one day any doctor around the globe can do this treatment ,but how long do you suppose will this take to become areality

    • EyeOnTheCure says:


      At this juncture, it is difficult to predict when a gene therapy for retinal conditions (including Stargardt disease) might be FDA approved and brought to the marketplace. Part of the challenge in making that prediction is that we don’t have a clinical-trial track record for gene therapies, because the treatment approach is so new. With that said, the companies (and researchers) developing these treatments are doing whatever they can to expedite the process, because they want to get treatments out to the patients (and generate revenue) as soon as they can. I am sure the companies have some idea or prediction for when they will be available, but are not at liberty to publicly divulge that information.

  9. Paul Aspland says:

    My daughter has stargardts and I monitor the various trials in the hope of news. Agree that safety is essential and core to developments, however is it not possible to have any indicative statement relating to the vision of the trial participants to date ?. (e.g “trial participants have experianced some improvement”). I guess the fact that the trial is ongoing indicates some visual improvement has been achieved ?.

    • EyeOnTheCure says:


      At this point, we don’t know whether emerging treatments (e.g., stem cells, gene therapies) for Stargardt disease will save vision, restore vision, or in some cases, both. It will likely take a few years to detect a treatment’s saving of vision – comparing vision loss in the treated eye versus the untreated eye. In most cases, Stargardt disease progresses slowly, so it takes time to determine if a potential treatment is having an effect. Furthermore, the early safety studies involve relatively small numbers of people, so we must be careful not to draw conclusions until a larger population of patients is treated. Many sponsors of clinical trials are very conservative about announcing results — they won’t until they feel confident that the results are real and sustained. On the other hand, some companies are quick to report results for a small number of patients, which may not reflect the treatment’s true long-term effect for most people.

      I think it is very appropriate to feel hopeful about these clinical trials, but we must be patient, as well.

  10. Judy Nelson says:

    I have a 27 year old daughter with Stargardt’s. We too are waiting for someone to tell us how many more years we have to wait. We are so grateful for all the research going on but when you have to watch your daughter’s vision slowly get worse I just want to hear someone say it will be X numbers of years and then we will have help for her. I pray every day for research and our scientists working on this dreadful disease. Thanks for listening!!! Judy

    • EyeOnTheCure says:

      Judy, thanks for your comment. As you said, we cannot know exactly when needed treatments and cures will come but we are more and more hopeful everyday! Progress is being made!

  11. Ashley says:

    I’m so glad to hear this good news. I understand the importance nance of safety first. I am 26 years old, I was diagnosed with stargardts 5 years ago. I pray that one day there is a cure for those going through this, i know its not easy! ‘m extremely grateful for all those amazing scientist working towards a cure, thank you all!

  12. Courtney says:

    I was diagnosed at 14 with stargardts Disease. i am now 23 and a mother of 2. I hope my children dont have this and assoon as any updates are available I’d love to know. Was told to take lutein beta caroteneand vitamins C and E. I was having slight improvements could be something to try while we wait!

    • EyeOnTheCure says:

      Courtney, thanks for commenting and sharing your story with us. As far as updates, definitely stay tuned to us and our emails as we are often providing research updates. If you have not already, please register on our website to start getting our emails. You may register at

      We would like to point you to a few places on our website that will provide some useful information on Stargardt Disease. Be sure to visit our section on this disease:

      There is a section on this page that discusses research advancements. While there is no cure for RP, much research is being done.

      We often report on new research advancements and so we encourage you to check out our Stargardts written articles information

      We hope you find this information helpful. Please let us know if there is anything else we can help you with.

    • Marc Peden says:

      Hello Courtney,
      Vitamin supplementation is a common recommendation however, I would please encourage you to actually avoid beta-carotene.
      As you may know, beta-carotene is a vitamin A derivative , and vitamin A is the building block for the visual proteins in the eye. In Stargardt’s disease, the problem lies in a faulty gene that normally helps in the recycling of these proteins. Because of the defective gene, these proteins back up and then have a tendency to produce a toxic waste product known a A2E. This is the waste product that builds up in the back of the eye in patients with Stargardt’s disease eventually leading to damage to the vision cells known as photoreceptors.
      By taking supplemental vitamin A, you may theoretically be providing more fuel to the fire. In fact, some more recent, promising clinical trials have actually looked at medications that slow the visual recycling process thereby perhaps slowing the accumulation of these waste products.
      Biochemically, I don’t see any other contraindication to the other supplements and recommend these to my Stargardt’s patients. The more recent AREDS2 vitamins are a good supplement to consider as they contain the lutein, vit C and E without beta-carotene.

      Hang in there as this is an exciting time for hereditary retinal disorders. There will be wonderful treatments that become available during your lifetime!

  13. Abe says:

    Hi there, thanks for your efforts. My wife was 9 yes old when stargardt’s disease set in. She is now 20, with an acuity of 20/400. We are expecting a baby now, hoping our baby doesn’t get it, also hope a cure is found.

  14. Adalina says:

    I’ve been affected by Stargardt’s since age 7; I don’t remember ever seeing well. Now at 15, I’m so grateful that all these people have dedicated their time to others like me… the hope that I might really be able to see everyone I love as more than a blob is incredible. Thank you so much!

    I’d also love to trade stories with anyone

  15. Sterling says:

    Hello to every , as I am truly eager of reading this website’s post to be updated on a regular basis.
    It contains pleasant stuff.

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  17. Connie says:

    When wil this treatment b available and wil it restore lost vision ?

  18. chaminda says:


    Thank You for your great job.My daughter is also suffering from stragardt,s disease .please let me know the updates

    Thank you

  1. […] company Sanofi has been granted broadened global development and commercialization rights for two retinal disease gene therapies: StarGen™, for Stargardt disease, and UshStat®, for Usher syndrome type 1B. Sanofi is expected […]

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