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Developing new therapies for Batten disease

Objective

The goal of BATCure is to advance the development of new therapeutic options for a group of rare lysosomal diseases - neuronal ceroid lipofuscinoses (NCL) or Batten disease. There are > thousand affected across Europe, with a combined incidence of c.1:100 000. The NCLs are devastating and debilitating genetic disorders that mainly affect children, who suffer progressive dementia and motor decline, visual failure and epilepsy, leading to a long period of complete dependence on others, and eventually a premature death. Existing palliative treatment can reduce, but does not eliminate, the burden of seizures and the progressively worsening effects on the whole body due to decreasing CNS influence and control. There are no curative treatments in the clinic for any type of NCL. We will follow a novel integrated strategy to identify specific gene and small molecule treatments for three genetic types of Batten disease that include the most prevalent world-wide, juvenile CLN3 disease, and in southern and mediterranean Europe, CLN6 and CLN7 diseases.

To develop new therapies for these 3 types of Batten disease, BATCure will:
1. Create new models, tools and technologies for developing and testing therapies
2. Further delineate disease biology and gene function to identify new therapeutic target pathways utilising yeast and pluripotent stem cell models
3. Identify biochemical therapeutic target pathways, facilitate effective evaluation of preclinical therapies and improve diagnostics
4. Extend a comprehensive natural history beyond the brain to include cardiology, the spinal cord, PNS, psychiatric and metabolic changes
5. Identify new and repurpose existing small molecule therapy
6. Triage new compound treatments in zebrafish, a high-throughput small vertebrate model
7. Deliver and monitor new treatments using mouse models
8. Provide a novel mechanism to involve patients and their families to inform and fully contribute to therapy development and prepare for clinical trials
Leaflet | Map data © OpenStreetMap contributors, Credit: EC-GISCO, © EuroGeographics for the administrative boundaries

Coordinator

UNIVERSITY COLLEGE LONDON

Address

Gower Street
Wc1e 6bt London

United Kingdom

Activity type

Higher or Secondary Education Establishments

EU Contribution

€ 1 873 453,75

Participants (13)

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AcureOmics AB

Sweden

EU Contribution

€ 314 375

LATVIJAS ORGANISKAS SINTEZES INSTITUTS

Latvia

EU Contribution

€ 399 625

CARDIFF UNIVERSITY

United Kingdom

EU Contribution

€ 223 966,25

FONDAZIONE TELETHON

Italy

EU Contribution

€ 566 247,50

UNIVERSITAETSKLINIKUM HAMBURG-EPPENDORF

Germany

EU Contribution

€ 815 350

PRONEXUS ANALYTICAL AB

Sweden

EU Contribution

€ 300 641,25

THE MANCHESTER METROPOLITAN UNIVERSITY

United Kingdom

EU Contribution

€ 224 707,50

THE ROYAL VETERINARY COLLEGE

United Kingdom

EU Contribution

€ 104 212,50

UNIVERSIDAD DE SALAMANCA

Spain

EU Contribution

€ 400 000

ORPHAZYME APS

Denmark

EU Contribution

€ 289 250

KING'S COLLEGE LONDON

United Kingdom

EU Contribution

€ 207 640

ACONDICIONAMIENTO TARRASENSE ASSOCIACION

Spain

EU Contribution

€ 213 962,50

BATTEN DISEASE FAMILY ASSOCIATION

United Kingdom

EU Contribution

€ 62 337,50

Project information

Grant agreement ID: 666918

Status

Closed project

  • Start date

    1 January 2016

  • End date

    30 June 2019

Funded under:

H2020-EU.3.1.3.

  • Overall budget:

    € 6 000 681,25

  • EU contribution

    € 5 995 768,75

Coordinated by:

UNIVERSITY COLLEGE LONDON

United Kingdom

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