Focus: Batten-1
Batten Disease
Batten disease (CLN3) is a rare, inherited neurodegenerative disorder that primarily affects children. A mutation in the CLN3 gene leads to the build-up of toxic substances (glycosphingolipids, or GSL) in the brain and retina. Over time, this accumulation causes progressive and irreversible damage to neurons, resulting in severe visual and neurological impairment.
Disease Progression
1. Early Stage
Symptoms typically appear between ages 4 and 6, often beginning with rapid vision loss that may lead to blindness.
2. Cognitive decline
Between ages 6 and 8, children experience progressive cognitive deterioration, leading to juvenile dementia.
3. Loss of autonomy
From ages 10 to 12, patients experience seizures, and a progressive loss of their motor and speech abilities.
4. Disease prognosis
The disease progresses steadily, significantly impacting quality of life and independence, and most patients do not survive beyond early adulthood.
Across the Globe
The juvenile form of Batten disease affects approximately 2,000 children worldwide. Mutations identified to date are predominantly found in patients of Scandinavian descent. The disease is more common in Northern Europe – particularly in Scandinavian countries, Germany, the United Kingdom, the Netherlands, Poland, and Baltic states – as well as in the United States, Canada, Australia, and New Zealand.
To date, there is no treatment for this form of the disease.
Our drug candidate: Batten-1
Batten-1 is the first drug candidate designed to slow – and potentially halt – disease progression. Its clinical potential has already been evaluated in patients, and it is now entering phase 3 trial, the final stage before potential regulatory approval and public access.
​« For families affected by Batten disease, Batten‑1 represents more than just a treatment – it is a real source of hope. After years of waiting, we are finally seeing a drug that could truly change the course of the disease, preserve abilities, connections, and time. Each step forward with Batten‑1 brings our children closer to a future where life is no longer defined by the disease, but by what they are still able to achieve. »
T. Craig Benson
Chair of Beyond Batten Disease Foundation
