GT038

**GT038** (also known as RhoNova) is an investigational gene therapy developed for the treatment of rhodopsin (RHO)-linked autosomal dominant retinitis pigmentosa (adRP), a rare inherited retinal dystrophy caused by dominant mutations in the RHO gene leading to progressive vision loss and blindness. It employs adeno-associated virus (AAV) vectors to deliver RNA interference (RNAi) molecules that suppress expression of both mutant and wild-type RHO alleles, while simultaneously providing a replacement wild-type RHO gene engineered to evade suppression, aiming to restore normal rhodopsin function in photoreceptor cells. Orphan Drug Designation has been granted by the FDA and EMA, with no approved pharmacologic treatments currently available for this condition affecting approximately 30,000 patients worldwide. Originally developed by Genable Technologies (founded by Professor Jane Farrar at Trinity College Dublin), it advanced through preclinical stages with investments supporting clinical trial preparation; Spark Therapeutics collaborated from 2014 (providing AAV manufacturing and expertise) and later acquired Genable, integrating GT038 into its inherited retinal disease pipeline.[1][3][7][8][13][17][18]

Other names

RhoNova