Thelansis

  Inherited retinal diseases (IRDs) possess a genetic origin, rendering them favorable candidates for RNA therapeutics treatment. Retinitis pigmentosa type 11 (RP11) is an autosomal dominant IRD among these conditions. Symptoms of RP11 typically manifest in early childhood, as patients experience a gradual decline in visual function and functional vision. By age 40, many RP11 patients find themselves in a state of legal blindness. Unfortunately, effective treatments for RP11 are currently absent. RP11 is categorized as a haploinsufficiency disorder, signifying that one copy of the relevant gene (in this case, the critical gene called PRPF31) operates normally. In contrast, the other copy carries a mutation that prevents the production of a properly functioning protein. In the context of RP11, this genetic imbalance results in insufficient protein levels within the retina, ultimately culminating in retinal disease. For haploinsufficiency conditions like RP11, RNA therapeutics offe...