RST-001

RST-001 is an investigational gene therapy developed to restore vision in patients with retinal degenerative diseases such as retinitis pigmentosa. It uses a non-replicating recombinant adeno-associated virus (AAV) vector to deliver the gene encoding channelrhodopsin-2 (ChR2) protein to retinal ganglion cells. Once expressed, ChR2 acts as a light-sensitive ion channel, enabling these cells to respond to light and transmit visual signals to the brain even after photoreceptor loss. This optogenetic approach is genotype-independent and aims to provide functional vision regardless of the underlying genetic defect causing photoreceptor degeneration[1][3][5][6]. The therapy has received orphan drug designation for retinitis pigmentosa and has completed Phase I/II clinical trials[2][8].