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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

Field of science

  • /medical and health sciences/medical biotechnology/cells technologies/stem cells
  • /medical and health sciences/basic medicine/neurology/epilepsy
  • /medical and health sciences/clinical medicine/cardiology
  • /humanities/history and archaeology/history

Call for proposal

H2020-PHC-2015-two-stage
See other projects for this call

Funding Scheme

RIA - Research and Innovation action

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)

AcureOmics AB
Sweden
EU contribution
€ 314 375
Address
Tvistevagen 48
SE-90187 Umeå
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
LATVIJAS ORGANISKAS SINTEZES INSTITUTS
Latvia
EU contribution
€ 399 625
Address
Aizkraukles 21
1006 Riga
Activity type
Research Organisations
CARDIFF UNIVERSITY
United Kingdom
EU contribution
€ 223 966,25
Address
Newport Road 30-36
CF24 ODE Cardiff
Activity type
Higher or Secondary Education Establishments
FONDAZIONE TELETHON
Italy
EU contribution
€ 566 247,50
Address
Via Varese 16/B
00185 Roma
Activity type
Research Organisations
UNIVERSITAETSKLINIKUM HAMBURG-EPPENDORF
Germany
EU contribution
€ 815 350
Address
Martinistrasse 52
20251 Hamburg
Activity type
Higher or Secondary Education Establishments
PRONEXUS ANALYTICAL AB
Sweden
EU contribution
€ 300 641,25
Address
Fogdevreten 2A
17177 Stockholm
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
THE MANCHESTER METROPOLITAN UNIVERSITY
United Kingdom
EU contribution
€ 224 707,50
Address
Oxford Road All Saints Building
M15 6BH Manchester
Activity type
Higher or Secondary Education Establishments
THE ROYAL VETERINARY COLLEGE
United Kingdom
EU contribution
€ 104 212,50
Address
Royal College Street
NW1 OTU London
Activity type
Higher or Secondary Education Establishments
UNIVERSIDAD DE SALAMANCA
Spain
EU contribution
€ 400 000
Address
Calle Patio De Escuelas 1
37008 Salamanca
Activity type
Higher or Secondary Education Establishments
ORPHAZYME APS
Denmark
EU contribution
€ 289 250
Address
Ole Maaloes Vej 3
2200 Kobenhavn
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
KING'S COLLEGE LONDON
United Kingdom
EU contribution
€ 207 640
Address
Strand
WC2R 2LS London
Activity type
Higher or Secondary Education Establishments
ACONDICIONAMIENTO TARRASENSE ASSOCIACION
Spain
EU contribution
€ 213 962,50
Address
Carrer De La Innovacio 2
08225 Terrassa
Activity type
Research Organisations
BATTEN DISEASE FAMILY ASSOCIATION
United Kingdom
EU contribution
€ 62 337,50
Address
4 Boundary Road
GU14 6SF Farnborough
Activity type
Other