Until Spark Therapeutics’ pioneering gene therapy, Luxturna, came along, patients with retinitis pigmentosa had few treatment options. Even after it was approved, though, the majority were left with the exact same options. Because it’s targeting mutations in a specific gene known as RPE65, Luxturna can only address 2 to 3% of the entire RP population. And they have $52 million-plus (€44.5 million) to realize that vision steadily over the next four or five years, with plans to complete both first- and second-in-human trials. What the founding scientists pinpointed, instead of a gene, was a neurotrophic factor secreted by rod photoreceptors that appears to protect the cones, which explains why RP patients see their cones die even in the absence of genetic abnormalities. By wrapping RdCVF, or rod-derived cone viability factor, and an oxidative stress reducing enzyme into a viral vector, they came up with SPVN06 in hopes of replacing one key function that RP patients lose alongside their rods.
For further information, see Sparing Vision (https://sparingvision.com/images/PDF/press_releases/2020/201021_SparingVision_financing.pdf) and ENDPOINTS NEWS (https://endpts.com/sparingvision-raises-52m-to-kick-off-long-journey-for-a-next-gen-gene-therapy-that-goes-much-much-broader-than-luxturna/)