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photoRetroSense Therapeutics develops game-changing gene therapy solutions to restore partial sight for certain eye diseases.

Frequently Asked Questions:

Are you currently in human clinical trials?

  • RetroSense is not in human trials at the time of writing. We are working to get into clinical trials quickly and safely.

How well will patients be able to see after treatment with RST-001?

  • Our hope – and expectation – is that we will be able to restore functional vision to patients. Until we have treated patients, we will not know exactly what level of vision will be restored. Animal models are encouraging, suggesting that we will be able to restore meaningful vision to patients who have been robbed of theirs by retinal degenerative disorders.

How is the treatment given?

  • This is a one-time injection into the eye. Such injections are now routine, outpatient procedures for most retina specialists. The gene is encapsulated in a virus “vector”. This vector has been proven safe in human clinical studies. It specializes in delivering genes (DNA) to the cells of interest. Once the gene is in the retinal cell, it begins to produce our light-sensitive protein.

How soon after injection will patients begin to see?

  • It takes some time for our light-sensitive protein to be “expressed” (produced from the gene). We expect expression to peak and level off by about 8 weeks. Early analysis suggests patients may experience some vision restoration in as little as 2 weeks, with improvements through about 8 weeks – corresponding to increases in light-sensitive protein levels. From that point, there may be additional processing that takes place between the retina and visual cortex, which would result in additional improvements in vision.

How long will the treatment last?

  • We expect the treatment to last many years – potentially for the life of the patient. Animal models with an ocular gene therapy approach are seeing benefit up to eight years, and counting.

Will there be a need for a "booster" shot?

  • While RetroSense does not anticipate the need for a booster shot, there may be follow-on treatments that could further enhance a patient’s vision. These are still early in development and would be separate therapies, altogether.

How long will treatment recipients have to stay in the hospital?

  • RetroSense anticipates this will be an outpatient procedure in the US.

What government or private institutions have supported the technology to date?

  • • RetroSense would like to thank all who have contributed to supporting the research and development of our channelrhodopsin-based approaches to vision restoration. To date several million has been infused at the university level and to RetroSense directly. Supporting institutions include:

  •     - Foundation Fighting Blindness / National Neurovision Research Institute
        - National Eye Institute at the National Institutes of Health
        - Michigan Pre-Seed Capital Fund
        - Wayne State University
        - Michigan Initiative for Innovation and Entrepreneurship
        - TechTown, Detroit
        - Qualifying Therapeutics Discovery Project
        - Ann Arbor Spark
        - Private investors

Is the gene to be injected derived from human stem cells?

  • No, it is a gene involved in photosensitivity in blue-green algae.

Who invented the treatment?

  • The pioneering work was performed by a group of scientists from Wayne State University in Michigan and Salus University in Pennsylvania, led by Dr. Zhuo-Hua Pan.

How do you know that the test animals became light sensitive?

  • In the initial study, WSU used mice with inherited blindness – models of retinitis pigmentosa. After treatment, light response patterns in their visual cortex were observed. Subsequently, rat models of retinitis pigmentosa were treated. Their behavior was observed, showing they responded to light signals in ways expected of sighted rats. Marmosets have also been treated, showing that the gene gets into the cells of interest and confers photosensitivity to those cells.

Will my rods & cones be restored to some form of working order?

  • The focus of our treatment is not on restoring rods and cones. Our objective is to make other retinal cells light sensitive. These cells remain in-place in retinitis pigmentosa – and they maintain a connection to the visual cortex.

Does your therapy target a specific genetic defect that causes RP, or does it have application across a broad range of RP?

  • We expect our proposed treatment to have application across a broad range of RP, irrespective of the specific genetic cause. This is because we target a feature common to all RP patients, which is the persistence of inner retinal cells after the loss of photoreceptors (rods and cones). RetroSense targets those cells, to make them light sensitive. Because those cells remain intact in all RP patients, we anticipate having application across all types of RP.

Is this like having an electronic implant put in my eye?

  • Both treatments are designed to restore vision. Our treatment will be far less invasive, requiring no implantation of electronic components. At present, retinal implants have a limited number of “pixels” – to the best of our knowledge, those currently in human trials have fewer than 100. RetroSense anticipates conferring photosensitivity to orders of magnitude more cells. Our expectation is that this will result in at least as good vision, if not better than that imparted by retinal implants.

I am only partially blind. Will RetroSense’s treatment improve my vision?

  • We will not know the full extent of vision restoration until we have tested the proposed treatment in humans. We are designing our clinical trials to yield as much information as possible, so that we will be able treat as many patients as will benefit from the treatment.

I understand that the development of new proposed treatments is expensive and time-consuming. What can I do to help?



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