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:
- Create new models, tools and technologies for developing and testing therapies
- Further delineate disease biology and gene function to identify new therapeutic target pathways utilising yeast and pluripotent stem cell models
- Identify biochemical therapeutic target pathways, facilitate effective evaluation of preclinical therapies and improve diagnostics
- Extend a comprehensive natural history beyond the brain to include cardiology, the spinal cord, PNS, psychiatric and metabolic changes
- Identify new and repurpose existing small molecule therapy
- Triage new compound treatments in zebrafish, a high-throughput small vertebrate model
- Deliver and monitor new treatments using mouse models
- Provide a novel mechanism to involve patients and their families to inform and fully contribute to therapy development and prepare for clinical trials
LEITAT’s role in the project:
WP1 New models Development, validation and optimisation of essential new models and tools.
- Generation of monoclonal antibodies. Immunize, screen, produce and purify high affinity monoclonal antibodies against CLN3, CLN6, CLN7 and for SCMAS. Leitat holds an extensive experience in the identification of new and key biomarkers and targets in the oncology area. Moreover, its experience and expertise in the generation of new mAb has led to the progression up to phase II
- Clinical trials of one of the antibodies generated in the past for Merck-Serono, and to the inclusion of several diagnostic antibodies in the Millipore catalogue.
- Identifying therapeutic target pathways, and developing new diagnostic and monitoring techniques using metabonomics.
- Profiling of microvesicles from vitro/vivo models and human samples by analyzing the miRNA expression profile and their proteome. Leitat has wide knowledge in the identification of new and key biomarkers and translate them on blood-based diagnostics, prognostics, and therapeutic guidance to support esearch activities and clinical practice, thus several patents has been filled.
Number of Contract: H2020/666918
Coordinator: UNIVERSITY COLLEGE LONDON
Email LEITAT manager in charge: Max Viallon
Starts: 1st of January 2016
Duration: 36 months
|LATVIJAS ORGANISKAS SINTEZES INSTITUTS||Latvia|
|CARDIFF UNIVERSITY||United Kingdom|
|PRONEXUS ANALYTICAL AB||Sweden|
|THE MANCHESTER METROPOLITAN UNIVERSITY||United Kingdom|
|THE ROYAL VETERINARY COLLEGE||United Kingdom|
|UNIVERSIDAD DE SALAMANCA||Spain|
|KING’S COLLEGE LONDON||United Kingdom|
|BATTEN DISEASE FAMILY ASSOCIATION||United Kingdom|