Our gene therapy is designed for one-time administration to yield potentially therapeutic levels of the missing enzyme tripeptidyl peptidase 1 (TPP1)
TPP1 is a lysosomal enzyme that in healthy neurons breaks down proteins in parts of the cell called a lysosome.
LTS-101 is designed to deliver functional TPP1 back to neurons and restore normal lysosomal function.
TPP1 is a lysosomal enzyme that in healthy neurons breaks down proteins in parts of the cell called a lysosome.
CLN2 disease (neuronal ceroid lipofuscinosis type 2), a form of Batten disease, is a rare, pediatric-onset, genetic disorder caused by an inherited mutation, or error, in the CLN2 gene. The CLN2 gene contains instructions to make an enzyme called tripeptidyl peptidase 1, or TPP1. TPP1 helps to break down and remove waste material from cells in the body. Deficiency in TPP1 enzyme activity causes waste materials to build up in the body’s cells, particularly in the brain and the eye, where they cause progressive damage and can lead to seizures, rapid loss of language and motor function, cognitive decline, vision loss/blindness, and premature death.
Clinical onset with presentation of seizures
Progressive onset of additional symptoms at typically 2 years after diagnosis
Phenotypical progression of development delay, seizures, motor decline blindness and death
“I really hope we can find a way to slow down this disease. No parent wants to see their kid go through endless hospital visits, the pain of treatments, or spend their days in a hospital bed. We just want more happy, normal days at home, more laughter, and less tears—more time being a family without the constant stress of medical issues.”
“Huntngton's Disease remains a devastating, incurable genetic brain disease, for which we desperately need effective interventions. A breakthrough would be transformative for affected patients and families worldwide.”
