Using gene therapy to slow or halt the progression
of neurodegenerative and neurodevelopmental disorders
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.
Many LSDs are caused by defects in the genes that encode lysosomal enzymes, normally responsible for breaking down and eliminating cellular waste products.
Lysogene’s gene-therapy technology involves introducing a normal copy of the affected gene, thereby enabling production of a functional version of the defective enzyme. This approach has the potential to slow, or even stop, disease progression.
gene therapy products have already been approved
human gene therapy clinical trials have been reported worldwide
OUR BEST-IN-CLASS TECHNOLOGY
Lysogene’s therapies focus on lysosomal storage diseases that affect the central nervous system (CNS). Our approach ensures efficient, safe delivery of the corrected gene to CNS cells.