RetroSense Therapeutics is pioneering an innovative gene therapy approach to restore vision in people with retinal degenerative conditions where the loss of photoreceptors has led to blindness. Photoreceptors (rods and cones) are light sensitive cells in the eye that convert light signals to nerve impulses which are sent to the brain where they are interpreted and create the vision we see. As photoreceptors die in retinal degenerative conditions such as retinitis pigmentosa (RP) and dry AMD, visual acuity and the ability to see in dimly lit rooms is diminished. Progressive loss of photoreceptors leads to blindness.
RetroSense is employing a gene therapy approach to deliver a new photosensitivity gene to retinal cells to restore the ability of eyes to sense light. As RP is caused by over 100 different gene defects, addressing each individually is not feasible with current technologies. RetroSense’s approach is designed to “install” new photosensors, restoring vision irrespective of which gene defect is responsible for vision loss. What this means is our approach promises application across a broad spectrum of RP patients.
RetroSense’s lead candidate, RST-001 employs a photosensitivity gene, channelrhodopsin-2, to create new photosensors in retinal cells and restore vision in retinal degenerative conditions such as RP and advanced dry-AMD.
Channelrhodopsin-2 is supported by a strong body of published literature on its efficacy and safety in animal models.1 Numerous studies have demonstrated the ability of channelrhodopsin-2 to restore light perception and vision in animals with naturally occurring or induced blindness due to loss of photoreceptors. In primate studies, the administration of channelrhodopsin-2 was well tolerated. This approach to vision restoration was pioneered by Dr. Zhuo-Hua Pan at Wayne State University and Dr. Alex Dizhoor at Salus University. 2
RetroSense is currently at the pre-clinical stage of development and working toward human clinical trials. RST-001 will be developed initially for retinitis pigmentosa, with advanced dry-AMD as a follow-on indication. We are targeting late 2013 to early 2014 to begin clinical trials. Please visit us periodically to see how we are making progress toward first-in-human studies.
1. Ivanova E, Hwang GS, Pan ZH, Troilo D. (May 2010). “Evaluation of AAV-Mediated Expression of Chop2-GFP in the Marmoset Retina.” Invest Ophthalmol Vis Sci.
2. Bi A, Cui J, Ma YP, “et al.” (April 2006). "Ectopic expression of a microbial-type rhodopsin restores visual responses in mice with photoreceptor degeneration." Neuron 50 (1): 23-33.