Freeline publishes proof-of-concept data for FLT190 to treat Fabry disease

Freeline Therapeutics announced the publication of preclinical proof-of-concept data for its gene therapy candidate for Fabry disease, FLT190, in the Nature journal Gene Therapy. Fabry disease is a debilitating genetic disorder in which an enzyme deficiency leads to a harmful build-up of fat in the cells that causes progressive organ damage and can result in early death."People living with Fabry disease can experience debilitating symptoms and disease progression, even with current treatments. There is a pressing need for therapies with more lasting efficacy that are less burdensome on Fabry families," said Pamela Foulds, M.D., Chief Medical Officer at Freeline. "We are encouraged by these preclinical data that support our ongoing Phase 1/2 clinical trial of FLT190 and our belief in FLT190 as a potential life-changing, one-time therapy for people with Fabry disease."