TreatRP: Translating cGMP analogues into a treatment for retinitis pigmentosa

Retinitis pigmentosa (RP) is characterised by loss of those cells in the eye that respond to light. These cells are called photoreceptors. Most forms of RP are today untreatable, and while individually they are rare, together they constitute a major cause of vision loss and blindness in the working age population. Because RP can be caused by mutations in more than 90 different genes, it is important to target common processes so that patients with different mutations may benefit from the same treatment. The goal of this proposal is, in fact, the development of a drug and treatment protocol for different forms of RP. The partners in this consortium previously found that high levels of the signalling molecule cyclic guanosine-3’-5’-mono-phosphate (cGMP) in photoreceptors cause cell death and consequently blindness. In previous collaborations they developed a new compound and delivery system, LP-CN03, which delayed the degenerative process and preserved photoreceptors in three animal models of RP. Now, TreatRP proposes to further advance the pre-clinical development of this promising approach, so that it will be ready for a clinical trial at the end of the project. To this end TreatRP aims at the pharmaceutical development of LP-CN03 to obtain a compound that meets all the requirements for the use in the clinic. This includes the study of the targets in photoreceptors of the new drug, as well as its possible toxicity, and finally the design of the first-in-man clinical trial protocol to enable an efficient and rapid translation of the new treatment to RP patients.