#	Pagina
attuale pagina	/open-h2020/projects/199719/index.html

Opendata, web and dolomites

BATCure SIGNED

Developing new therapies for Batten disease

Total Cost €

EC-Contrib. €

Partnership

Views

Outcomes and
results

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.

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

Project "BATCure" data sheet

The following table provides information about the project.

Coordinator	UNIVERSITY COLLEGE LONDON Organization address address: GOWER STREET 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	http://www.batcure.eu
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

Partnership

Take a look of project's partnership.

#	participants	country	role	EC contrib. [€]
1	UNIVERSITY COLLEGE LONDON UNIVERSITY COLLEGE LONDON Organization address address: GOWER STREET 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.	UK (LONDON)	coordinator	1˙873˙453.00
2	UNIVERSITAETSKLINIKUM HAMBURG-EPPENDORF UNIVERSITAETSKLINIKUM HAMBURG-EPPENDORF Organization address address: Martinistrasse 52 city: HAMBURG postcode: 20251 website: www.uke.uni-hamburg.de contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	DE (HAMBURG)	participant	815˙350.00
3	FONDAZIONE TELETHON FONDAZIONE TELETHON Organization address address: VIA VARESE 16/B city: ROMA postcode: 185 website: www.telethon.it contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	IT (ROMA)	participant	566˙247.00
4	UNIVERSIDAD DE SALAMANCA UNIVERSIDAD DE SALAMANCA Organization address address: CALLE PATIO DE ESCUELAS 1 city: SALAMANCA postcode: 37008 website: www.usal.es contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	ES (SALAMANCA)	participant	400˙000.00
5	LATVIJAS ORGANISKAS SINTEZES INSTITUTS LATVIJAS ORGANISKAS SINTEZES INSTITUTS Organization address address: AIZKRAUKLES 21 city: RIGA postcode: 1006 website: www.osi.lv contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	LV (RIGA)	participant	399˙625.00
6	AcureOmics AB AcureOmics AB Organization address address: TVISTEVAGEN 48 city: Umeå postcode: SE-90187 website: www.acureomics.com contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	SE (Umeå)	participant	314˙375.00
7	PRONEXUS ANALYTICAL AB PRONEXUS ANALYTICAL AB Organization address address: FOGDEVRETEN 2A city: STOCKHOLM postcode: 17177 website: www.pronexus.se contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	SE (STOCKHOLM)	participant	300˙641.00
8	ORPHAZYME APS ORPHAZYME APS Organization address address: OLE MAALOES VEJ 3 city: KOBENHAVN postcode: 2200 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.	DK (KOBENHAVN)	participant	289˙250.00
9	THE MANCHESTER METROPOLITAN UNIVERSITY THE MANCHESTER METROPOLITAN UNIVERSITY Organization address address: OXFORD ROAD ALL SAINTS BUILDING city: MANCHESTER postcode: M15 6BH website: www.mmu.ac.uk contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	UK (MANCHESTER)	participant	224˙707.00
10	CARDIFF UNIVERSITY CARDIFF UNIVERSITY Organization address address: NEWPORT ROAD 30-36 city: CARDIFF postcode: CF24 ODE website: www.cardiff.ac.uk contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	UK (CARDIFF)	participant	223˙966.00
11	ACONDICIONAMIENTO TARRASENSE ASSOCIACION ACONDICIONAMIENTO TARRASENSE ASSOCIACION Organization address address: CARRER DE LA INNOVACIO 2 city: TERRASSA postcode: 8225 website: www.leitat.org contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	ES (TERRASSA)	participant	213˙962.00
12	KING'S COLLEGE LONDON KING'S COLLEGE LONDON Organization address address: STRAND city: LONDON postcode: WC2R 2LS website: www.kcl.ac.uk contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	UK (LONDON)	participant	207˙640.00
13	THE ROYAL VETERINARY COLLEGE THE ROYAL VETERINARY COLLEGE Organization address address: ROYAL COLLEGE STREET city: LONDON postcode: NW1 OTU website: www.rvc.ac.uk contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	UK (LONDON)	participant	104˙212.00
14	BATTEN DISEASE FAMILY ASSOCIATION BATTEN DISEASE FAMILY ASSOCIATION Organization address address: 4 BOUNDARY ROAD city: FARNBOROUGH postcode: GU14 6SF 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.	UK (FARNBOROUGH)	participant	62˙337.00

Map

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

Deliverables

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.

Publications

List of publications.
year	authors and title	journal	last update
2019	Alfried KohlschÃ¼tter, 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. BolaÃ±os 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 Î±-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 KÃ¤kelÃ¤, 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, SÃ¸ren Holm, Erik Nord, Jeff Richardson, Silvio Garattini, Peter Kolominsky-Rabas, Deborah Marshall, Ulf Persson, Maarten Postma, Steven Simoens, Oriol de SolÃ Morales, Keith Tolley, Mondher Toumi, Harry Telser, James R Bonham, Helmut Hintner, Anja Diem, Martin Laimer, RÃ©jean HÃ©bert, 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. BolaÃ±os, Tristan R. McKay NF-ÎºB 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 Oâ€™Leary, AndrÃ© S L M Antunes, Nuria Palomar, Patrick Aldrin-Kirk, Mark Basche, Antonette Bennett, Zelpha Dâ€™Souza, HÃ©lÃ¨ne 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 ClÃ©ment, Tomas BjÃ¶rklund, 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. BolaÃ±os, 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 Niemannâ€“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. BolaÃ±os 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, MaÃ«lle 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		2019-08-29

Are you the coordinator (or a participant) of this project? Plaese send me more information about the "BATCURE" project.

For instance: the website url (it has not provided by EU-opendata yet), the logo, a more detailed description of the project (in plain text as a rtf file or a word file), some pictures (as picture files, not embedded into any word file), twitter account, linkedin page, etc.

Send me an email (fabio@fabiodisconzi.com) and I put them in your project's page as son as possible.

Thanks. And then put a link of this page into your project's website.

The information about "BATCURE" are provided by the European Opendata Portal: CORDIS opendata.