GAMUT’s lead product, now SPVN20, is a novel, mutation-agnostic gene therapy, which aims at restoring the function of dormant cone cells in the retina.
If approved it would compete with Novartis’ Luxturna (voretigene neparvovec), the gene therapy from Roche/Spark Therapeutics, but with a much broader indication.
Luxturna only works in people with inherited retinal disease due to mutations in both copies of the RPE65 gene, about 2% of patients with the condition.
SPVN20 is an AAV vector-based product encoding for a variant of GIRK, a GPCR activated ion channel that could restore sight in patients regardless of the gene that causes their RP.
