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Genetic and Small Molecule Modifiers of Lysosomal Function

Objective

LysoMod will innovate in the area of personalized medicine for disorders linked to lysosomal dysfunction. This will be achieved by implementing a collaborative staff-exchange program between highly complementary and multidisciplinary academic and non-academic partners with expertise in pharmacology, medicinal chemistry, cell biology, biochemistry, mouse and human genetics, transcriptomics, proteomics and lipidomics. Based on the critical role that lysosomes play in cells, a better understanding of lysosomal function will have a major impact on human health, fostering the development of new strategies to improve quality of life for people affected by a variety of diseases, ranging from lysosomal storage diseases (LSDs) to age-related neurodegenerative disorders. LysoMod’s specific objectives are: 1) to develop and further optimize existing therapies for LSDs; 2) to identify new targets for personalized therapies for LSDs; and 3) to investigate the cross-talk between lysosomal function, signalling pathways and gene expression regulation. The pioneer work of a participant in the consortium led to the development of a drug that is approved for clinical use. LysoMod will i) investigate the mechanisms of action of this and other drugs in lysosome-related disorder; ii) identify modifier genes involved in LSD pathology and test their potential as new targets for personalized therapeutic approaches; iii) identify candidate RNAs that can be targeted to enhance lysosomal function. The companies in the consortium will ensure a rapid transfer of new knowledge into applications for diagnostics and clinical trials. Prioritising lysosomal dysfunction as a highly relevant biomedical problem, the LysoMod consortium will implement a mentored staff-exchange program to provide young researchers with high-level training in innovative approaches for exploring biological systems, preparing the next generation of researchers for careers either in the private or public health sectors.

Coordinator

INSTITUTO DE MEDICINA MOLECULAR JOAO LOBO ANTUNES
Net EU contribution
€ 343 800,00
Address
Avenida Prof Egas Moniz
1649 028 Lisboa
Portugal

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Region
Continente Área Metropolitana de Lisboa Área Metropolitana de Lisboa
Activity type
Research Organisations
Non-EU contribution
€ 0,00

Participants (9)

THE CHANCELLOR, MASTERS AND SCHOLARS OF THE UNIVERSITY OF OXFORD
United Kingdom
Net EU contribution
€ 207 000,00
Address
Wellington Square University Offices
OX1 2JD Oxford

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Region
South East (England) Berkshire, Buckinghamshire and Oxfordshire Oxfordshire
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 9 000,00
THE UNIVERSITY OF SUSSEX
United Kingdom
Net EU contribution
€ 81 000,00
Address
Sussex House Falmer
BN1 9RH Brighton

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Region
South East (England) Surrey, East and West Sussex Brighton and Hove
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
WEIZMANN INSTITUTE OF SCIENCE
Israel
Net EU contribution
€ 22 950,00
Address
Herzl Street 234
7610001 Rehovot

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Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 45 000,00
FONDAZIONE TELETHON ETS

Participation ended

Italy
Net EU contribution
€ 0,00
Address
Via Varese 16/b
00185 Roma

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Region
Centro (IT) Lazio Roma
Activity type
Research Organisations
Non-EU contribution
€ 0,00
INSTITUTUL DE BIOCHIMIE
Romania
Net EU contribution
€ 99 000,00
Address
Splaiul Independentei No. 296
060031 Bucharest

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Region
Macroregiunea Trei Bucureşti-Ilfov Bucureşti
Activity type
Research Organisations
Non-EU contribution
€ 0,00
LIPOTYPE
Germany
Net EU contribution
€ 4 500,00
Address
Tatzberg 47
01307 Dresden

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
Sachsen Dresden Dresden, Kreisfreie Stadt
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00
GENOMED, DIAGNOSTICOS DE MEDICINA MOLECULAR SA
Portugal
Net EU contribution
€ 276 750,00
Address
Avenida Professor Egas Moniz
1600 190 Lisboa

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
Continente Área Metropolitana de Lisboa Área Metropolitana de Lisboa
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00
CHRONOS BIOSYSTEMS SRL
Romania
Net EU contribution
€ 45 000,00
Address
Bulevardul Geniului, Nr 34, Parter, Camera 2
060117 Bucharest

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Region
Macroregiunea Trei Bucureşti-Ilfov Bucureşti
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00
INTRABIO LTD
United Kingdom
Net EU contribution
€ 22 500,00
Address
Summit House, 170 Finchley Road,
NW3 6BP London

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
London Inner London — West Camden and City of London
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00

Partners (4)

Partner

Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.

PONTIFICIA UNIVERSIDAD CATOLICA DE CHILE
Chile
Net EU contribution
€ 0,00
Address
Avenida Libertador Bernardo O'higgins 340
8331150 Santiago

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Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 148 950,00
Partner

Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.

UNIVERSIDAD DE BUENOS AIRES
Argentina
Net EU contribution
€ 0,00
Address
Calle Viamonte 430
1053 Buenos Aires

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Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 49 500,00
Partner

Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.

THE CHILDREN'S HOSPITAL OF PHILADELPHIA NON PROFIT ORG
United States
Net EU contribution
€ 0,00
Non-EU contribution
€ 54 000,00
Partner

Partner organisations contribute to the implementation of the action, but do not sign the Grant Agreement.

UNIVERSIDAD DEL DESARROLLO
Chile
Net EU contribution
€ 0,00
Address
Av La Plaza 700
San Carlos De Apoquindo

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Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 81 000,00