Translating cGMP analogues for the treatment of retinitis pigmentosa

Retinitis pigmentosa (RP) is characterized by loss of cells in the eye that respond to light, ie photoreceptor cells.

Most forms of RP are today untreatable, and while individually they are rare, together they constitute a major cause of vision loss 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 signaling molecule cyclic GMP in photoreceptors cause cell death and consequently blindness.

In previous collaborations, they developed a compound and delivery system (LP-CN03), that reduced the degenerative process and preserved photoreceptors in three animal models of RP.

Here, TreatRP proposes to advance the pre-clinical development of this promising approach, so that it will become ready for a clinical trial.

To this end TreatRP aims at the pharmaceutical development of LP-CN03 to obtain a compound that meets all requirements for the use in the clinic.

This includes the evaluation of the new drug’s targets in photoreceptors, 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.