Listen to this page using ReadSpeaker

ARVO 2015 Highlight: AMD Gene Therapy Performs Encouragingly in Human Study

an AMD eyeWhile treatments such as Lucentis®, Avastin®, and Eylea® have been saving and restoring vision for people with wet age-related macular degeneration (AMD) over the last several years, they have a significant drawback: The therapies require regular injections into the eye—in some cases, monthly—for the life of the patient.

But there was good news reported during ARVO’s annual meeting last week, about an early-stage clinical trial of a wet AMD gene therapy known as RetinoStat® that requires far fewer injections. While researchers are still learning about the long-term efficacy of the treatment, they believe one injection will last for several years, perhaps the lifetime of the patient.

Oxford BioMedica, the developer of RetinoStat, reported that the treatment was safe and expressed the desired therapeutic proteins during the 48-week, 21-patient, Phase I study. Most exciting, patients had stable visual acuity. Also, the vision-robbing, vascular leakage that’s the hallmark of wet AMD was reduced. The study results were selected as an ARVO meeting “Hot Topic.”

Phase I studies are primarily focused on safety, so there is a long way to go before Oxford BioMedica can conclude that the treatment works and seek regulatory approval. Many more patients need to be evaluated over longer time periods. But these results are encouraging.

With earlier, preclinical funding from the Foundation Fighting Blindness, Oxford BioMedica initiated commercial development of three retinal-disease gene therapies—RetinoStat as well as StarGen™, for Stargardt disease, and UshStat®, for Usher syndrome type 1B. In 2014, the international pharmaceutical company Sanofi acquired development and commercialization rights to StarGen and UshStat. These early-stage clinical trials are ongoing, and we’ll report results when the company makes them available.

Pictured, above: A retina affected by the hemorrhaging associated with wet AMD. Photo courtesy of the National Eye Institute. 


11 Responses to 'ARVO 2015 Highlight: AMD Gene Therapy Performs Encouragingly in Human Study'

  1. How can I be part of ARVO 2015 Highlight STUDY in Richmond VA with wet AMD????? My retinal doctors are Va. Eye Institute.

    • EyeOnTheCure says:

      You need to check the website: Clinicaltrials.gov that is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

  2. Lenny macek says:

    Are there any study’s going on for cone rod dystrophy??

    • EyeOnTheCure says:

      Approximately 50% of all cone-rod dystrophy (CRD) is caused by mutations in the ABCA4 gene, the same gene that is involved in Stargardt’s disease. If you have this gene mutation, then you might qualify for the ABCA4 gene therapy trial that is currently recruiting patients in Portland, Oregon and Paris, France. It should only take a month or two to find out if the ABCA4 gene is involved. If no mutation can be found in the ABCA4 gene, other candidate CRD genes will need to be screened. 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
      Whether you identify your disease gene or not, you should consider participating in FFB’s “My Retina Tracker”, a free registry that can help you find out about clinical trials that are recruiting for your specific disease. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

  3. bernard la fianza says:

    are you seeking study participants?

    • EyeOnTheCure says:

      To find out about clinical trials, you should check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information. You should also consider participating in FFB’s “My Retina Tracker”, a free registry that can help you find out about clinical trials that are recruiting for your specific disease. For more information on “My Retina Tracker” please see the following web link:
      https://www.myretinatracker.org/

    • sajad says:

      My son is sufferin from retinits pegmentosa by brith age 21.How can be i part of ARVO 2015.

  4. Bill Wittke says:

    please help me to get into this study

Leave a Reply

Your email address will not be published. Required fields are marked *

*