Portfolio of gene therapy treatment
Dimension’s programs address significant unmet medical needs for patients living with hemophilia and rare diseases associated with the liver. The Company’s lead program in hemophilia B is currently in phase I/II clinical testing. The company is also advancing programs through preclinical testing to address hemophilia A in collaboration with Bayer HealthCare and for patients living with ornithine transcarbamylase (OTC) deficiency and glycogen storage disease type Ia (GSDIa). Additional programs addressing citrullinemia type 1, phenylketonuria, and Wilson disease in candidate selection continue to broaden and expand our liver-directed rare disease platform.
Dimension’s therapies use the biology of the parvovirus (adeno-associated virus (AAV)) to carry the missing gene in the body to the target cell with the aim of correcting the defect and providing meaningful clinical benefit to the lives of patients. This platform has demonstrated early validation and compelling preclinical in vivo proof of concept across multiple disease states in leading academic research centers. / Learn More /