FLT190

**FLT190** is an investigational gene therapy developed for the treatment of Fabry disease, a rare lysosomal storage disorder caused by mutations in the GLA gene leading to deficient alpha-galactosidase A (α-Gal A) enzyme activity. FLT190 utilizes a proprietary adeno-associated virus vector (AAVS3) to deliver a functional GLA gene specifically to liver cells via intravenous administration. This enables sustained production and secretion of α-Gal A enzyme, aiming to reduce substrate accumulation (such as globotriaosylceramide, Gb3, and lyso-Gb3) in tissues. FLT190 was in Phase 1/2 clinical trials (MARVEL-1), showed increased α-Gal A activity and promising substrate reduction, but development was paused for strategic reasons in 2023 by Freeline Therapeutics (now under Syncona).