Gene therapy holds extraordinary promise in treating a range of human diseases. It is particularly well-suited to conditions that are linked to defects in a single gene.
Lysogene’s two lead programs focus on Lysosomal Storage Disorders (LSDs). LSDs are a group of rare, inherited conditions characterized by an abnormal build-up in cells of metabolic by-products such as fats, proteins, or sugars.
The accumulation of these products prevents the cells from functioning normally, causing a range of progressive symptoms among young children. Two– thirds of LSDs affect the central nervous system (CNS), impacting movement, cognitive ability, and the control of other physiological functions.