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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-SC1-2017-Two-Stage-RTD
See other projects for this call

Coordinator

ACADEMISCH ZIEKENHUIS LEIDEN
Address
Albinusdreef 2
2333 ZA Leiden
Netherlands
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 2 195 625

Participants (16)

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
EU contribution
€ 437 500
Address
Rue De Tolbiac 101
75654 Paris
Activity type
Research Organisations
UNIVERSITY COLLEGE LONDON
United Kingdom
EU contribution
€ 120 000
Address
Gower Street
WC1E 6BT London
Activity type
Higher or Secondary Education Establishments
GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST
United Kingdom
EU contribution
€ 57 500
Address
Great Ormond Street
WC1N 3JH London
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
OSPEDALE SAN RAFFAELE SRL
Italy
EU contribution
€ 532 500
Address
Via Olgettina 60
20132 Milano
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
MEDIZINISCHE HOCHSCHULE HANNOVER
Germany
EU contribution
€ 558 960
Address
Carl-neuberg-strasse 1
30625 Hannover
Activity type
Higher or Secondary Education Establishments
UNIVERSITAET ULM
Germany
EU contribution
€ 57 500
Address
Helmholtzstrasse 16
89081 Ulm
Activity type
Higher or Secondary Education Establishments
UNIWERSYTET MEDYCZNY IM PIASTOW SLASKICH WE WROCLAWIU
Poland
EU contribution
€ 57 500
Address
Ul Pasteura 1
50 367 Wroclaw
Activity type
Higher or Secondary Education Establishments
UNIVERSITY OF NEWCASTLE UPON TYNE
United Kingdom
EU contribution
€ 57 500
Address
Kings Gate
NE1 7RU Newcastle Upon Tyne
Activity type
Higher or Secondary Education Establishments
INTERNATIONAL PATIENT ORGANISATION FOR PRIMARY IMMUNODEFICIENCIES
United Kingdom
EU contribution
€ 82 500
Address
Main Road Firside
PL11 3LE Torpoint
Activity type
Other
BATAVIA BIOSCIENCES BV
Netherlands
EU contribution
€ 982 125
Address
Zernikedreef 16
2333 CL Leiden
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
FUNDACIO HOSPITAL UNIVERSITARI VALL D'HEBRON - INSTITUT DE RECERCA
Spain
EU contribution
€ 57 500
Address
Passeig Vall D Hebron 119-129 Edificio De Recerca
08035 Barcelona
Activity type
Research Organisations
OSPEDALE PEDIATRICO BAMBINO GESU
Italy
EU contribution
€ 57 500
Address
Piazza Sant Onofrio 4
00165 Roma
Activity type
Research Organisations
ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
France
EU contribution
€ 25 000
Address
3 Avenue Victoria
75000 Paris
Activity type
Research Organisations
MEDICAL RESEARCH INFRASTRUCTURE DEVELOPMENT AND HEALTH SERVICES FUND BY THE SHEBA MEDICAL CENTER
Israel
EU contribution
€ 57 500
Address
Tel Hashomer Sheba Medical Center
52621 Ramat Gan
Activity type
Research Organisations
LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN
Germany
EU contribution
€ 57 500
Address
Geschwister Scholl Platz 1
80539 Muenchen
Activity type
Higher or Secondary Education Establishments
GENEWERK GMBH
Germany
EU contribution
€ 596 250
Address
Im Neuenheimer Feld 582
69120 Heidelberg
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)