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Stem-cell based gene therapy for recombination deficient SCID (RECOMB)

Objective

Gene therapy for rare inherited immune disorders has become a clinical reality. Especially for SCID, two major types of SCID (ADA-SCID, X-SCID) have been successfully treated by autologous stem cell based gene therapy. However, for the most common group of SCID, the SCID underlying recombination defects, this has not yet occurred due to the higher complexities of the affected genes involved. The aim of the current proposal is to fill the unmet medical need for the most common major category of SCID, recombination activating gene-1 and -2 (RAG-1, -2) deficient SCID, by performing Stage I/II clinical trials using autologous hematopoietic stem cell based gene therapy . To this end we will develop novel safety assays, pre-GMP and GMP lentiviral batches and design and conduct multicenter, multinational clinical trials with input from regulatory authorities such as EMA and patient advocacy groups. The two trials will be conducted with phenotypic, molecular (integration sites, therapeutic gene expression) and functional readouts and should lead to effective treatment for > 70% of all SCID patients in Europe. RECOMB forms the logical extension of highly successful previous EU consortia that have made the EU global leader in gene therapy for orphan immune diseases.

Call for proposal

H2020-SC1-2016-2017

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Sub call

H2020-SC1-2017-Two-Stage-RTD

Coordinator

ACADEMISCH ZIEKENHUIS LEIDEN
Net EU contribution
€ 2 195 625,00
Address
Albinusdreef 2
2333 ZA Leiden
Netherlands

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Region
West-Nederland Zuid-Holland Agglomeratie Leiden en Bollenstreek
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00

Participants (16)

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
Net EU contribution
€ 437 500,00
Address
Rue De Tolbiac 101
75654 Paris

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Region
Ile-de-France Ile-de-France Paris
Activity type
Research Organisations
Non-EU contribution
€ 0,00
UNIVERSITY COLLEGE LONDON
United Kingdom
Net EU contribution
€ 120 000,00
Address
Gower Street
WC1E 6BT London

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Region
London Inner London — West Camden and City of London
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST
United Kingdom
Net EU contribution
€ 57 500,00
Address
Great Ormond Street
WC1N 3JH London

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Region
London Inner London — West Camden and City of London
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Non-EU contribution
€ 0,00
OSPEDALE SAN RAFFAELE SRL
Italy
Net EU contribution
€ 532 500,00
Address
Via Olgettina 60
20132 Milano

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Region
Nord-Ovest Lombardia Milano
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00
MEDIZINISCHE HOCHSCHULE HANNOVER
Germany
Net EU contribution
€ 558 960,00
Address
Carl-neuberg-strasse 1
30625 Hannover

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Region
Niedersachsen Hannover Region Hannover
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
UNIVERSITAET ULM
Germany
Net EU contribution
€ 57 500,00
Address
Helmholtzstrasse 16
89081 Ulm

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Region
Baden-Württemberg Tübingen Ulm, Stadtkreis
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
UNIWERSYTET MEDYCZNY IM PIASTOW SLASKICH WE WROCLAWIU
Poland
Net EU contribution
€ 57 500,00
Non-EU contribution
€ 0,00
UNIVERSITY OF NEWCASTLE UPON TYNE
United Kingdom
Net EU contribution
€ 57 500,00
Address
Kings Gate
NE1 7RU Newcastle Upon Tyne

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Region
North East (England) Northumberland and Tyne and Wear Tyneside
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
INTERNATIONAL PATIENT ORGANISATION FOR PRIMARY IMMUNODEFICIENCIES
United Kingdom
Net EU contribution
€ 82 500,00
Address
Main Road Firside
PL11 3LE Torpoint

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Region
South West (England) Cornwall and Isles of Scilly Cornwall and Isles of Scilly
Activity type
Other
Non-EU contribution
€ 0,00
BATAVIA BIOSCIENCES BV
Netherlands
Net EU contribution
€ 982 125,00
Address
Zernikedreef 16
2333 CL Leiden

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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
West-Nederland Zuid-Holland Agglomeratie Leiden en Bollenstreek
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,75
FUNDACIO HOSPITAL UNIVERSITARI VALL D'HEBRON - INSTITUT DE RECERCA
Spain
Net EU contribution
€ 57 500,00
Address
Passeig Vall D Hebron 119-129 Edificio De Recerca
08035 Barcelona

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Region
Este Cataluña Barcelona
Activity type
Research Organisations
Non-EU contribution
€ 0,00
OSPEDALE PEDIATRICO BAMBINO GESU
Italy
Net EU contribution
€ 57 500,00
Address
Piazza Sant Onofrio 4
00165 Roma

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Region
Centro (IT) Lazio Roma
Activity type
Research Organisations
Non-EU contribution
€ 0,00
ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
France
Net EU contribution
€ 25 000,00
Address
3 Avenue Victoria
75000 Paris

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Region
Ile-de-France Ile-de-France Paris
Activity type
Research Organisations
Non-EU contribution
€ 0,00
MEDICAL RESEARCH INFRASTRUCTURE DEVELOPMENT AND HEALTH SERVICES FUND BY THE SHEBA MEDICAL CENTER
Israel
Net EU contribution
€ 57 500,00
Non-EU contribution
€ 0,00
LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN
Germany
Net EU contribution
€ 57 500,00
Address
Geschwister Scholl Platz 1
80539 Muenchen

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Region
Bayern Oberbayern München, Kreisfreie Stadt
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
PROTAGENE CGT GMBH
Germany
Net EU contribution
€ 596 250,00
Address
Im Neuenheimer Feld 582
69120 Heidelberg

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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
Baden-Württemberg Karlsruhe Heidelberg, Stadtkreis
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00