CLN2 disease is a fatal neurodegenerative disease with no curative treatment

CLN2 disease (neuronal ceroid lipofuscinosis type 2), one of the 13 subtypes of Batten disease, is an ultra-rare, fatal neurodegenerative disorder affecting approximately 1 in 100,000 live births world-wide. It results from a lack or loss of function in the tripeptidyl peptidase 1 (TPP1) enzyme, which helps to break down and remove waste materials from neurons. 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.

CLN2 disease progresses relentlessly

At ~2.5 years old

Clinical onset with presentation of seizures

At 4-5 years old

Progressive onset of additional symptoms

At ~8-12 years old

Phenotypical progression of development delay, seizures, motor decline, blindness, and death

For more information, please visit the Batten Disease Support, Research, & Advocacy Foundation

Testimonials

Expert perspectives on transforming treatment in CLN2 disease

“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.”

—Caregiver

“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.”

—Caregiver

“HD 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.”

—HD Clinician

ABOUT LTS-101

Our gene therapy candidate is designed to potentially alter the treatment paradigm for CLN2 patients

CLN2 Disease

In CLN2 disease, TPP1 is mutated and cannot breakdown proteins. This causes material to accumulate in lysosomes, causing cellular distress and eventually neuronal death, leading to the symptoms of CLN2 disease.

TPP1 is a lysosomal enzyme that, in healthy neurons, breaks down proteins in parts of the cell called a lysosome

LTS-101 Mechanism of Action

LTS-101 is designed to deliver functional TPP1 back to neurons and restore normal lysosomal function

Better Capsid

LTS-101 exploits our AAV-Ep+ capsid to deliver TPP1 throughout the CNS

See our approach