About Fabry Disease
Fabry disease is a monogenic disease caused by mutations in the GLA gene which encodes for the alpha-galactosidase A (AGA) enzyme that results in the body’s inability to produce sufficient AGA enzyme activity, causing the accumulation of toxic levels of sphingolipids, such as the substrate globotriaosylceramide-3 (Gb3), in critical organs, including the heart, kidney and blood vessels. The cardiomyopathy in Fabry disease is the leading cause of death, accounting for 62% of deaths, and is secondary to the systemic lysosomal storage disease syndrome. We estimate the potential initial addressable male Fabry patient population in the United States and EU-5 to be up to 19,000 individuals, 57% of which suffer from classic Fabry disease.
Learn more about Fabry disease and watch a video provided by the American Society of Gene and Cell Therapy organization which
highlights this unmet need in the heart.
Our lead product candidate, 4D-310, is currently in an ongoing Phase 1/2 clinical trial in adult patients with classic (severe) Fabry disease. The primary endpoint of this trial is to determine the safety and maximum-tolerated dose. Secondary endpoints include biomarker assessments of plasma AGA activity and markers of biologic activity in the heart, including cardiac MRI. We expect to treat early onset classic Fabry disease patients initially and hope to expand into late-onset patients, including those with cardiomyopathy.
4D-310 received Fast Track Designation from the FDA in third quarter of 2020. For more information about 4DMT’s Phase 1/2 Trial of 4D-310 in adult patients with classic (severe) Fabry disease, please visit Clinicaltrials.gov (NCT04519749).