Lysosomal Therapeutics Inc. (LTI) is dedicated to innovative, research and development in the field of neurodegeneration. Our strategy leverages the genetically and clinically validated link between lysosomal disorders and neurodegenerative diseases to establish a unique and effective platform for novel drug discovery.
We harness our world-class knowledge of lysosomal biology, enzymology, and drug development to deliver innovative therapies for severe neurological diseases. Our lead program builds on the genetic association between Parkinson’s disease (PD) and the GBA1 gene. This GBA1 gene codes for the glucocerebrosidase (GCase) enzyme, which is critical for optimal lipid processing in the lysosomal compartment of the cell. A mistake or mutation in one copy of the GBA1 gene significantly increases the lifetime risk of developing PD, and also affects the subsequent rate of disease progression. GBA1 mutations are now known to be the most common genetic risk factor for parkinsonism, accounting for approximately 10% of cases in the U.S. and Europe 1.
The company has developed LTI-291, a novel, first-in-class drug compound aimed to specifically treat patients with GBA-associated parkinsonism (GBA-AP). Additionally, LTI is expanding its research into other lysosomal enzyme deficiencies, which may be significantly involved in multiple neurodegenerative diseases, thus unlocking the true potential of the company’s discovery platform.
LTI is led by a team of seasoned scientists, clinical professionals and business leaders who have extensive experience in drug discovery and development, focused on genetic and neurodegenerative diseases. Our team is highly motivated to make a real difference as soon as possible for patients with severe neurodegenerative and neurodevelopmental diseases.
1. Liu, G., Boot, B., Locascio, J. J., Jansen, I. E., Winder-Rhodes, S., Eberly, S., Elbaz, A., Brice, A., Ravina, B., van Hilten, J. J., Cormier-Dequaire, F., Corvol, J.-C., Barker, R. A., Heutink, P., Marinus, J., Williams-Gray, C. H., Scherzer, C. R. and for the International Genetics of Parkinson Disease Progression (IGPP) Consortium (2016), Specifically neuropathic Gaucher's mutations accelerate cognitive decline in Parkinson's. Ann Neurol., 80: 674–685. doi:10.1002/ana.24781