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Promising Research Highlighted at Meeting of Retinal Gene Therapy Experts

genesWhen it comes to fighting blinding retinal diseases, nothing has been more exciting than the advancement of gene therapies into clinical trials. And, with human studies of gene therapies now underway for Leber congenital amaurosis (LCA, RPE65 mutations), Usher syndrome type 1B, Stargardt disease, retinoschisis, choroideremia and retinitis pigmentosa (MERTK mutations), scientists are gaining new knowledge every day about the best technologies and methodologies for gene-therapy development.

To share knowledge of the successes, opportunities and challenges of this promising treatment approach for the broad spectrum of retinal conditions, the Casey Eye Institute, Oregon Health & Science University (OHSU), recently hosted a meeting of 130 of the world’s leading retinal gene-therapy experts. The one-day event was supported in part by the Foundation Fighting Blindness and included 27 presentations, many of which focused on clinical development.

The Foundation embraces these collaborative events to facilitate the exchange of valuable information between investigators, who are often so busy with their own projects they don’t have time to share knowledge with their colleagues. Also, the information shared often has universal applicability. What we learn from one study, even if it’s directed toward a specific disease, often moves the entire gene-therapy field forward.

The meeting featured presentations on many different topics, including current clinical trials, surgical and imaging techniques and natural-history studies. Below are highlights of three projects, which have strong applicability to most gene-therapy development efforts.

Robin Ali, Ph.D., University College London, reported results for his team’s 12-patient Phase I/II clinical trial of a gene therapy for people with LCA (RPE65). He reported that many study participants showed improved retinal sensitivity, night vision and mobility. However, the treatment didn’t slow retinal degeneration, and the vision benefits were not sustained in the long-term. Dr. Ali and his colleagues concluded that humans need more RPE65 protein than previously studied LCA animal models to prevent degeneration. As a result, the investigators have made a new, more potent RPE65 gene therapy, and plan to launch another human trial by the end of 2015. Results of this study underscore the limitations in correlating human and animal data and the challenges of determining a safe, optimal treatment dose for patients.

Paul Yang, M.D., Ph.D., OHSU, provided an update on the 18-patient, Phase I/II UshStat gene-therapy clinical trial for people with Usher syndrome type 1B, which is caused by mutations in the gene MYO7A. Three doses of the therapy will be administered. Patients as young as 6 years old may receive the therapy. Six patients have been treated thus far with two different doses. No significant adverse events have been reported. While safety is the primary focus of the trial, the investigators are conducting several tests and imaging studies to determine if the therapy slows retinal degeneration. Because MYO7A is a relatively large gene, the therapy uses a lentivirus for gene delivery. It has a larger cargo capacity than most gene therapies in human studies, which use adeno-associated viruses or AAVs. Researchers are interested in comparing safety and effectiveness of AAVs and lentiviruses, and which do a better job of gene delivery to different types of retinal cells.

One of the challenges of evaluating potential therapies in clinical trials is that a therapy’s effect on vision can be difficult to measure in a short time. In many cases, it can take several years to capture vision reduction. Also, there can be significant variation in test results for the same patient, even when tested on the same day. Richard Weleber, M.D., Ph.D., discussed a technique called “Modeling the Hill of Vision,” which provides a detailed, three-dimensional image of a patient’s visual field, the portion of the retina that’s functional. He generates visual-field information and exports it to a computer program to generate the three-dimensional image, which captures exquisite details of vision and structural changes. Dr. Weleber has used the technique for several human studies, including those for RPE65 gene therapy, valproic acid, StarGen (Stargardt disease gene therapy) and UshStat.

While there was a lot of great news that came out of the gene therapy meeting, it’s clear there’s much more work that needs to be done to bring an end to all retinal diseases—whether through gene therapy or other treatment approaches. But thanks to our amazingly innovative and talented scientists, and the sustained support of the Foundation’s generous donors, we will achieve our goal. It is no doubt challenging, but we have the passion and commitment to do it.


17 Responses to 'Promising Research Highlighted at Meeting of Retinal Gene Therapy Experts'

  1. majid says:

    I hope one day a quire will be found for the sake of my 13 year old daughter
    god bless you all.

  2. Akhil Bhatia says:

    after reading I too devloped confidence that there are chances cure for psuedomonas which has resulted in eye vision loss in my right eye kindly do help me please

  3. Rosa Capone says:

    I just received news that my 17 year old son has stargardts disease and I hope your developments and studies not only helps him soon, but so many children that need your help. Please if you have any latest help for his disease please Please email me. Thank you all and God Bless you for helping people.

    • Chad Mayfield says:

      I was diagnosed with Stargardts when I was 10 years old. I am now 38 years old. In less than 28 years the once impossible has turned into hope. What I have learned in these 28 years is it isn’t your abilities or in some cases disabilities that define you as a person but the journey and the attitude that nothing will stop me from achieving my dreams. You find a way to adapt and it makes you stronger, a better person, and appreciative of what you have. This is a long and challenging lesson to learn for a young adult but I am sure your child has/will see this experience in a similar light. Committed and passionate doctors give us hope. And with hope we can achieve the once impossible.

  4. Ashish says:

    I am 35 year old male and I just come to know about stargardts disease 3 months back. Please email e if you have any latest help about this disease.

    • EyeOnTheCure says:

      You will be interested to know that the Foundation Fighting Blindness is partnering with Sanofi to conduct a gene therapy clinical trial for Stargardt disease in Oregon and Paris, France. For more information on this trial, see the following link:
      http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2
      Before you can participate in any gene therapy trial you must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf
      I highly recommend that you enroll in “My Retina Tracker”, a free registry that can help people find out about clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/
      You may be interested to know that there is a “Stargardt – Macular Degeneration” Facebook page where you can communicate with other families affeced by Stargardt disease. Here is the link:
      https://www.facebook.com/groups/Stargardts/

      Finally, here is a list of pharmaceutical / biotech companies that are developing therapies for Stargardt Disease:

      Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.

      Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
      *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

      Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660
      Visum Therapeutics, (http://www.visumtherapeutics.com) Visum Therapeutics is developing a small molecule therapy to treat Stargardt’s disease. Visum’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Visum has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Visum has identified a lead compound, VSM 20R, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Visum plans to conduct Phase I and Phase II clinical trials in the near future.

      I hope you find this information helpful. Please feel free to contact me if you have any other questions or concerns.

  5. Carol says:

    Our 19.5 yr old daughter was diagnosed with Stargardts in Jan 2015. I hope that one day very soon all this research will become a cure. We are desperate for a cure! A pre-med student with such a bright future ahead of her and in her 2nd year of college, is inconceivable to think she will be limited without a cure. Pleas help!

    • EyeOnTheCure says:

      I am sorry to hear of your daughter’s diagnosis. Your daughter and you will be pleased to know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. For more information on this trial, see the following link:
      http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2
      Before your daughter can apply to participate in the Sanofi or any other gene therapy trial she must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf
      I also highly recommend that your daughter enrolls in “My Retina Tracker”, a free registry that helps people find out about clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/
      Your daughter may also be interested to know that there is a “Stargardt – Macular Degeneration” Facebook page where she can communicate with other people affected by Stargardt disease. Here is the link: https://www.facebook.com/groups/Stargardts/
      Finally, below is a list of pharmaceutical / biotech companies that are developing therapies for Stargardt Disease:

      Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.

      Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
      *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

      Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660
      Visum Therapeutics, (http://www.visumtherapeutics.com) Visum Therapeutics is developing a small molecule therapy to treat Stargardt’s disease. Visum’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Visum has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Visum has identified a lead compound, VSM 20R, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Visum plans to conduct Phase I and Phase II clinical trials in the near future.
      I hope you find this information helpful. Please feel free to contact me if you have any other questions or concerns.

  6. Chaminda says:

    Hi

    My daughter is suffering with stargardts disease . If you know new updates please let me know .I wish the success for your research which will help not only my daughter but the others as well
    Thank You

  7. Robyn says:

    Our daughter just diagnosed with Stargart’s disease. Wonderful to hear positivity and hope for a cure. Is there any way to get regular information?

    • EyeOnTheCure says:

      Please stay tuned into the Foundation’s website for updated information. You may already know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. For more information on this trial, see the following link:
      http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2 However, before your daughter can apply to participate in any gene therapy trial she must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/pages/pdfs/Genetic-Testing-Booklet-V5.2-20151023.pdf
      I also highly recommend that your daughter enrolls in “My Retina Tracker”, a free registry that helps people find out about clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/
      Your daughter may also be interested to know that there is a “Stargardt – Macular Degeneration” Facebook page where she can communicate with other people affected by Stargardt disease. Here is the link: https://www.facebook.com/groups/Stargardts/
      Finally, below is a list of pharmaceutical / biotech companies that are developing therapies for Stargardt Disease:
      Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.
      Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
      *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.
      Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660
      Visum Therapeutics, (http://www.visumtherapeutics.com) Visum Therapeutics is developing a small molecule therapy to treat Stargardt’s disease. Visum’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Visum has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Visum has identified a lead compound, VSM 20R, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Visum plans to conduct Phase I and Phase II clinical trials in the near future.

  8. Maria Pereira says:

    Both my kids aged 17 and 9 are diagnosed of Stargardt.
    I am sure with the progress of stem cell therapy there has to be a solution to help our children. Please keep me updated about any progress.
    Thank you

    • EyeOnTheCure says:

      I am sorry to hear of your children’s diagnosis. You will be pleased to know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. For more information on this trial, see the following link:
      http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2 However, before your children can participate in any gene therapy trial they must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf
      You should also consider enrolling your children in “My Retina Tracker”, a free registry that helps people find out about clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/
      Your will also be interested to know that there is a “Stargardt – Macular Degeneration” Facebook page where you and your children can communicate with other people affected by Stargardt disease. Here is the link: https://www.facebook.com/groups/Stargardts/

      Below is a list of pharmaceutical / biotech companies that are developing therapies for Stargardt Disease:

      Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.

      Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
      *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

      Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660
      Vision Medicine (Previously Visum) The Foundation Fighting Blindness is partnering with Vision Medicine to develop a small molecule therapy Stargardt disease. Vision Medicine’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Vision Medicine has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Vision Medicine has identified a lead compound, VM 200, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Vision Medicine plans to conduct Phase I and Phase II clinical trials in the near future. To read more about the partnership between Vision Medicine and FFB, see the following web link:
      http://www.blindness.org/foundation-news/foundation-fighting-blindness-partners-vision-medicines-develop-stargardt-disease

  9. Raghu says:

    I wish the clinical trials on humans are successful and FDA approves this as treatment mechanism. I am confident that this method will be cure for Stargardt’s.

    Thank you.

  10. Richard Shortall says:

    I am a 21 year old man that has been living with stargardt for the last 9 years with 20/200 vision. It’s been a struggle coping and learning to live with this disease, but it helps knowing there’re people dedicated to finding a cure. Thank you and please email me with any updates.

    • EyeOnTheCure says:

      Dear Sir, you will be pleased to know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. For more information on this trial, see the following link:
      http://clinicaltrials.gov/ct2/show/NCT01367444?term=stargardt&recr=Open&no_unk=Y&rank=2 However, before anyone can apply for participation in a gene therapy trial, they must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document: http://www.blindness.org/sites/default/files/genetic_testing_booklet_201311rev.pdf

      Anyone with a retinal disease should consider enrolling in “My Retina Tracker”, a free registry that helps link people with retinal disease to appropriate clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link: https://www.myretinatracker.org/

      Of general interest, there is a “Stargardt – Macular Degeneration” Facebook page where people can communicate with other people affected by Stargardt disease. Here is the link: https://www.facebook.com/groups/Stargardts/

      Below is a list of pharmaceutical / biotech companies that are developing therapies for Stargardt Disease:

      Acucela,( http://www.acucela.com/) is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.

      Ocata Therapeutics (https://www.ocata.com/), a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page: http://www.clinicaltrials.gov/ct2/show/NCT01469832?term=advanced+cell+technology&rank=2
      *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

      Alkeus, (http://alkeus.com/) Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical trials.gov recruitment page: https://www.clinicaltrials.gov/ct2/show/NCT02402660
      Vision Medicine (Previously Visum) The Foundation Fighting Blindness is partnering with Vision Medicine to develop a small molecule therapy Stargardt disease. Vision Medicine’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Vision Medicine has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Vision Medicine has identified a lead compound, VM 200, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Vision Medicine plans to conduct Phase I and Phase II clinical trials in the near future. To read more about the partnership between Vision Medicine and FFB, see the following web link:
      http://www.blindness.org/foundation-news/foundation-fighting-blindness-partners-vision-medicines-develop-stargardt-disease

      I hope you find this information helpful. Please feel free to contact me if you have any other questions or concerns.Thank you for your support that is enabling the development of new treatments for degenerative retinal disease.

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