Lysosomal Storage Disease Product Pipeline

About Fabry Disease

Fabry disease is a progressive and fatal condition with cardiac complications cited as the leading cause of death. We estimate the total addressable male Fabry patient population in the US to be approximately 10,000 individuals. The approved enzyme replacement therapies (ERT) reportedly do not adequately address Fabry cardiomyopathy, a leading cause of death in this disease.

4DMT Pipeline Product: 4D-310

Our lead product candidate in lysosomal storage diseases is wholly-owned by 4DMT: 4D-310 under development for the treatment of Fabry disease. 4D-310 uses a proprietary vector designed for efficient, low dose intravenous delivery to cardiac and other relevant tissues and to have minimal toxicities, and potential resistance to pre-existing antibodies in the human population.

In 2019 we initiated IND-enabling pharmacology and Good Laboratory Practices (GLP) toxicology and biodistribution studies with 4D-310, and we completed pre-IND communications with the U.S. Food and Drug Administration (FDA). 4D-310 Phase 1/2 clinical trial initiation is planned for the second half of 2020.

Patient Natural History Study
In the beginning of 2020, we expect to initiate a natural history study in patients with Fabry disease. Patients will be followed over time for changes in biomarkers of Fabry disease, including cardiac MRI (to assess substrate build up and cardiomyopathy), blood levels of AGA and the substrate Gb3, and organ function. We expect some of these patients will subsequently enroll in our planned Phase 1/2 clinical trial of 4D-310.