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Developing new therapies for Batten disease

Total Cost €


EC-Contrib. €






 BATCure project word cloud

Explore the words cloud of the BATCure project. It provides you a very rough idea of what is the project "BATCure" about.

cns    progressively    group    pns    incidence    batten    repurpose    gt    mediterranean    prevalent    therapies    monitor    follow    biochemical    zebrafish    yeast    inform    tools    evaluation    brain    suffer    cord    visual    complete    debilitating    gene    clinical    combined    models    devastating    strategy    batcure    mainly    ncl    lysosomal    options    vertebrate    cln6    reduce    disease    seizures    clinic    throughput    function    families    body    model    ncls    mouse    ceroid    diseases    neuronal    cln7    cln3    triage    dementia    patients    dependence    history    palliative    worsening    mechanism    lipofuscinoses    prepare    curative    trials    juvenile    period    technologies    types    pluripotent    metabolic    stem    decreasing    diagnostics    treatment    preclinical    epilepsy    genetic    decline    cardiology    spinal    thousand    influence    disorders    children    natural    delineate    utilising    involve    world    treatments    southern    progressive    molecule    death    extend    create    therapy    small    psychiatric    biology    burden    rare    compound    motor    eliminate    therapeutic    premature    cell   

Project "BATCure" data sheet

The following table provides information about the project.


Organization address
city: LONDON
postcode: WC1E 6BT
website: n.a.

contact info
title: n.a.
name: n.a.
surname: n.a.
function: n.a.
email: n.a.
telephone: n.a.
fax: n.a.

 Coordinator Country United Kingdom [UK]
 Project website
 Total cost 6˙000˙681 €
 EC max contribution 5˙995˙768 € (100%)
 Programme 1. H2020-EU.3.1.3. (Treating and managing disease)
 Code Call H2020-PHC-2015-two-stage
 Funding Scheme RIA
 Starting year 2016
 Duration (year-month-day) from 2016-01-01   to  2019-06-30


Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    UNIVERSITY COLLEGE LONDON UK (LONDON) coordinator 1˙873˙453.00
3    FONDAZIONE TELETHON IT (ROMA) participant 566˙247.00
4    UNIVERSIDAD DE SALAMANCA ES (SALAMANCA) participant 400˙000.00
6    AcureOmics AB SE (Umeå) participant 314˙375.00
7    PRONEXUS ANALYTICAL AB SE (STOCKHOLM) participant 300˙641.00
8    ORPHAZYME APS DK (KOBENHAVN) participant 289˙250.00
10    CARDIFF UNIVERSITY UK (CARDIFF) participant 223˙966.00
12    KING'S COLLEGE LONDON UK (LONDON) participant 207˙640.00
13    THE ROYAL VETERINARY COLLEGE UK (LONDON) participant 104˙212.00


 Project 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


List of deliverables.
Host NCL- 2018 Websites, patent fillings, videos etc. 2020-02-17 16:44:29
Provide range of reports across different media for dissemination of the final results of the project Documents, reports 2020-02-17 16:43:56
Report of feedback analysis and its dissemination to key stakeholders Documents, reports 2020-02-17 16:43:48
Key resources translated into relevant major EU languages Documents, reports 2020-02-17 16:43:48
First internal report on gender issues Documents, reports 2019-07-26 14:35:25
Project kick-off meeting minutes Documents, reports 2019-07-26 14:35:23
Dissemination poster and leaflets Documents, reports 2019-07-26 14:35:24
Research key target groups required for clincal trial development Documents, reports 2019-07-26 14:35:25
Set up BATCure web site Websites, patent fillings, videos etc. 2019-07-26 14:35:23
Website pages, facebook & twitter feeds established for all stakeholders & wider public Documents, reports 2019-07-26 14:35:24

Take a look to the deliverables list in detail:  detailed list of BATCure deliverables.


year authors and title journal last update
List of publications.
2019 Alfried Kohlschütter, Angela Schulz, Udo Bartsch, Stephan Storch
Current and Emerging Treatment Strategies for Neuronal Ceroid Lipofuscinoses
published pages: 315-325, ISSN: 1172-7047, DOI: 10.1007/s40263-019-00620-8
CNS Drugs 33/4 2020-01-28
2019 Olga Burmistrova, Ana Olias-Arjona, Rebeca Lapresa, Daniel Jimenez-Blasco, Tatiana Eremeeva, Dmitry Shishov, Sergei Romanov, Kristina Zakurdaeva, Angeles Almeida, Peter O. Fedichev, Juan P. Bolaños
Targeting PFKFB3 alleviates cerebral ischemia-reperfusion injury in mice
published pages: , ISSN: 2045-2322, DOI: 10.1038/s41598-019-48196-z
Scientific Reports 9/1 2019-11-15
2019 Emyr Lloyd-Evans, Helen Waller-Evans
Lysosomal Ca 2+ Homeostasis and Signaling in Health and Disease
published pages: a035311, ISSN: 1943-0264, DOI: 10.1101/cshperspect.a035311
Cold Spring Harbor Perspectives in Biology 2019-11-15
2018 Giulia Massaro, Citra N. Z. Mattar, Andrew M. S. Wong, Ernestas Sirka, Suzanne M. K. Buckley, Bronwen R. Herbert, Stefan Karlsson, Dany P. Perocheau, Derek Burke, Simon Heales, Angela Richard-Londt, Sebastian Brandner, Mylene Huebecker, David A. Priestman, Frances M. Platt, Kevin Mills, Arijit Biswas, Jonathan D. Cooper, Jerry K. Y. Chan, Seng H. Cheng, Simon N. Waddington, Ahad A. Rahim
Fetal gene therapy for neurodegenerative disease of infants
published pages: 1317-1323, ISSN: 1078-8956, DOI: 10.1038/s41591-018-0106-7
Nature Medicine 24/9 2019-11-15
2018 Eridan Rocha-Ferreira, Laura Poupon, Aura Zelco, Anna-Lena Leverin, Syam Nair, Andrea Jonsdotter, Ylva Carlsson, Claire Thornton, Henrik Hagberg, Ahad A Rahim
Neuroprotective exendin-4 enhances hypothermia therapy in a model of hypoxic-ischaemic encephalopathy
published pages: 2925-2942, ISSN: 0006-8950, DOI: 10.1093/brain/awy220
Brain 141/10 2019-11-15
2017 F. Bellomo, D. L. Medina, E. De Leo, A. Panarella, F. Emma
High-content drug screening for rare diseases
published pages: 601-607, ISSN: 0141-8955, DOI: 10.1007/s10545-017-0055-1
Journal of Inherited Metabolic Disease 40/4 2019-11-15
2019 Jonathan Lambert, Steven Howe, Ahad Rahim, Derek Burke, Simon Heales
Inhibition of Mitochondrial Complex I Impairs Release of α-Galactosidase by Jurkat Cells
published pages: 4349, ISSN: 1422-0067, DOI: 10.3390/ijms20184349
International Journal of Molecular Sciences 20/18 2019-11-15
2019 Yevgeniya Atiskova, Susanne Bartsch, Tatyana Danyukova, Elke Becker, Christian Hagel, Stephan Storch, Udo Bartsch
Mice deficient in the lysosomal enzyme palmitoyl-protein thioesterase 1 (PPT1) display a complex retinal phenotype
published pages: , ISSN: 2045-2322, DOI: 10.1038/s41598-019-50726-8
Scientific Reports 9/1 2019-11-15
2019 Lisa von Kleist, Khandsuren Ariunbat, Ingke Braren, Tobias Stauber, Stephan Storch, Tatyana Danyukova
A newly generated neuronal cell model of CLN7 disease reveals aberrant lysosome motility and impaired cell survival
published pages: 196-205, ISSN: 1096-7192, DOI: 10.1016/j.ymgme.2018.09.009
Molecular Genetics and Metabolism 126/2 2019-08-29
2019 Carolin Schmidtke, Stephan Tiede, Melanie Thelen, Reijo Käkelä, Sabrina Jabs, Georgia Makrypidi, Marc Sylvester, Michaela Schweizer, Ingke Braren, Nahal Brocke-Ahmadinejad, Susan L. Cotman, Angela Schulz, Volkmar Gieselmann, Thomas Braulke
Lysosomal proteome analysis reveals that CLN3-defective cells have multiple enzyme deficiencies associated with changes in intracellular trafficking
published pages: 9592-9604, ISSN: 0021-9258, DOI: 10.1074/jbc.ra119.008852
Journal of Biological Chemistry 294/24 2019-08-29
2016 Michael Schlander, Søren Holm, Erik Nord, Jeff Richardson, Silvio Garattini, Peter Kolominsky-Rabas, Deborah Marshall, Ulf Persson, Maarten Postma, Steven Simoens, Oriol de Solà Morales, Keith Tolley, Mondher Toumi, Harry Telser, James R Bonham, Helmut Hintner, Anja Diem, Martin Laimer, Réjean Hébert, Nabarun Dasgupta, Carrie E. Pierce, Melissa Jordan, Barbara Bori, Mohanad Fors, Emilie Prazak
8th European Conference on Rare Diseases & Orphan Products (ECRD 2016)
published pages: , ISSN: 1750-1172, DOI: 10.1186/s13023-016-0515-y
Orphanet Journal of Rare Diseases 11/S1 2019-08-29
2019 Sara E Mole, Glenn Anderson, Heather A Band, Samuel F Berkovic, Jonathan D Cooper, Sophia-Martha Kleine Holthaus, Tristan R McKay, Diego L Medina, Ahad A Rahim, Angela Schulz, Alexander J Smith
Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis
published pages: 107-116, ISSN: 1474-4422, DOI: 10.1016/s1474-4422(18)30368-5
The Lancet Neurology 18/1 2019-08-29
2018 Sophia-Martha kleine Holthaus, Joana Ribeiro, Laura Abelleira-Hervas, Rachael A. Pearson, Yanai Duran, Anastasios Georgiadis, Robert D. Sampson, Matteo Rizzi, Justin Hoke, Ryea Maswood, Selina Azam, Ulrich F.O. Luhmann, Alexander J. Smith, Sara E. Mole, Robin R. Ali
Prevention of Photoreceptor Cell Loss in a Cln6 Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar Cells
published pages: 1343-1353, ISSN: 1525-0016, DOI: 10.1016/j.ymthe.2018.02.027
Molecular Therapy 26/5 2019-08-29
2017 Juliette M. K. M. Delhove, Suzanne M. K. Buckley, Dany P. Perocheau, Rajvinder Karda, Patrick Arbuthnot, Neil C. Henderson, Simon N. Waddington, Tristan R. McKay
Longitudinal in vivo bioimaging of hepatocyte transcription factor activity following cholestatic liver injury in mice
published pages: , ISSN: 2045-2322, DOI: 10.1038/srep41874
Scientific Reports 7/1 2019-08-29
2017 Rajvinder Karda, Dany P. Perocheau, Natalie Suff, Joanne Ng, Juliette M. K. M. Delhove, Suzanne M. K. Buckley, Samantha Richards, John R. Counsell, Henrik Hagberg, Mark R. Johnson, Tristan R. McKay, Simon N. Waddington
Continual conscious bioluminescent imaging in freely moving somatotransgenic mice
published pages: , ISSN: 2045-2322, DOI: 10.1038/s41598-017-06696-w
Scientific Reports 7/1 2019-08-29
2018 Lorna M. FitzPatrick, Kate E. Hawkins, Juliette M.K.M. Delhove, Emilio Fernandez, Chiara Soldati, Louise F. Bullen, Axel Nohturfft, Simon N. Waddington, Diego L. Medina, Juan P. Bolaños, Tristan R. McKay
NF-κB Activity Initiates Human ESC-Derived Neural Progenitor Cell Differentiation by Inducing a Metabolic Maturation Program
published pages: 1766-1781, ISSN: 2213-6711, DOI: 10.1016/j.stemcr.2018.03.015
Stem Cell Reports 10/6 2019-08-29
2018 Julie Tordo, Claire O’Leary, André S L M Antunes, Nuria Palomar, Patrick Aldrin-Kirk, Mark Basche, Antonette Bennett, Zelpha D’Souza, Hélène Gleitz, Annie Godwin, Rebecca J Holley, Helen Parker, Ai Yin Liao, Paul Rouse, Amir Saam Youshani, Larbi Dridi, Carla Martins, Thierry Levade, Kevin B Stacey, Daniel M Davis, Adam Dyer, Nathalie Clément, Tomas Björklund, Robin R Ali, Mavis Agbandje-M
A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency
published pages: 2014-2031, ISSN: 0006-8950, DOI: 10.1093/brain/awy126
Brain 141/7 2019-08-29
2018 L. Felipe Barros, Juan P. Bolaños, Gilles Bonvento, Anne-Karine Bouzier-Sore, Angus Brown, Johannes Hirrlinger, Sergey Kasparov, Frank Kirchhoff, Anne N. Murphy, Luc Pellerin, Michael B. Robinson, Bruno Weber
Current technical approaches to brain energy metabolism
published pages: 1138-1159, ISSN: 0894-1491, DOI: 10.1002/glia.23248
Glia 66/6 2019-08-29
2018 Michael P Hughes, Dave A Smith, Lauren Morris, Claire Fletcher, Alexandria Colaco, Mylene Huebecker, Julie Tordo, Nuria Palomar, Giulia Massaro, Els Henckaerts, Simon N Waddington, Frances M Platt, Ahad A Rahim
AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann–Pick type C1 disease
published pages: 3079-3098, ISSN: 0964-6906, DOI: 10.1093/hmg/ddy212
Human Molecular Genetics 27/17 2019-08-29
2018 Seila Fernandez-Fernandez, Veronica Bobo-Jimenez, Raquel Requejo-Aguilar, Silvia Gonzalez-Fernandez, Monica Resch, Monica Carabias-Carrasco, Joaquim Ros, Angeles Almeida, Juan P. Bolaños
Hippocampal neurons require a large pool of glutathione to sustain dendrite integrity and cognitive function
published pages: 52-61, ISSN: 2213-2317, DOI: 10.1016/j.redox.2018.08.003
Redox Biology 19 2019-08-29
2016 Rita J. Guerreiro, Rachel Brown, Donnai Dian, Christian de Goede, Jose Bras, Sara E. Mole
Mutation of TBCK causes a rare recessive developmental disorder
published pages: e76, ISSN: 2376-7839, DOI: 10.1212/NXG.0000000000000076
Neurology Genetics 2/3 2019-08-29
2018 Tatyana Danyukova, Khandsuren Ariunbat, Melanie Thelen, Nahal Brocke-Ahmadinejad, Sara E Mole, Stephan Storch
Loss of CLN7 results in depletion of soluble lysosomal proteins and impaired mTOR reactivation
published pages: 1711-1722, ISSN: 0964-6906, DOI: 10.1093/hmg/ddy076
Human Molecular Genetics 27/10 2019-08-29
2017 Sara E Mole
BATCure, Developing new therapies for Batten disease, Horizon2020
published pages: 37-39, ISSN: 2398-7073, DOI: 10.21820/23987073.2017.10.37
Impact 2017/10 2019-08-29
2018 Julien Baruteau, Dany P. Perocheau, Joanna Hanley, Maëlle Lorvellec, Eridan Rocha-Ferreira, Rajvinder Karda, Joanne Ng, Natalie Suff, Juan Antinao Diaz, Ahad A. Rahim, Michael P. Hughes, Blerida Banushi, Helen Prunty, Mariya Hristova, Deborah A. Ridout, Alex Virasami, Simon Heales, Stewen J. Howe, Suzanne M. K. Buckley, Philippa B. Mills, Paul Gissen, Simon N. Waddington
Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer
published pages: , ISSN: 2041-1723, DOI: 10.1038/s41467-018-05972-1
Nature Communications 9/1 2019-08-29
2017 Davide Marotta, Elisa Tinelli, Sara E. Mole
NCLs and ER: A stressful relationship
published pages: 1273-1281, ISSN: 0925-4439, DOI: 10.1016/j.bbadis.2017.04.003
Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease 1863/6 2019-08-29
2019 kleine Holthaus SM, Herranz-Martin S, Massaro G, Aristorena M, Hoke J, Hughes MP, Maswood R, Semenyuk O, Basche M, Shah AZ, Klaska IP, Smith AJ, Mole SE, Rahim AA, Ali RR
Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease
published pages: , ISSN: , DOI: 10.1101/673848

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