OliX Pharmaceuticals Inc. in South Korea, a leading developer of RNAi therapeutics, is expanding its research in ocular diseases. The company recently added a new program, OLX304A, which aims to develop an RNAi therapy to treat retinitis pigmentosa (RP). RNAi therapy is a relatively new approach that identifies disease targets that can be inhibited or shut down as a potential treatment strategy. The company believes its technology is optimal for developing ocular therapeutics and that OLX304A will be an innovative approach to treating RP. This treatment should work for all RP patients regardless of their mutation-causing gene. The company hopes to begin Phase 1 clinical trials in 2023.
What this means for Usher syndrome: Because vision loss in Usher syndrome is caused by retinitis pigmentosa, this new RNAi therapy could expand the treatment options available to Usher patients. This potential therapy is still in its early stages, but if proven successful, could be beneficial broadly to Usher patients since it is not gene or mutation-specific.