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RECOMB SIGNED

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

Total Cost €

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EC-Contrib. €

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Partnership

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Project "RECOMB" data sheet

The following table provides information about the project.

Coordinator
ACADEMISCH ZIEKENHUIS LEIDEN 

Organization address
address: ALBINUSDREEF 2
city: LEIDEN
postcode: 2333 ZA
website: www.lumc.nl

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 Netherlands [NL]
 Project website http://www.recomb.eu
 Total cost 5˙990˙460 €
 EC max contribution 5˙990˙460 € (100%)
 Programme 1. H2020-EU.3.1.3. (Treating and managing disease)
 Code Call H2020-SC1-2017-Two-Stage-RTD
 Funding Scheme RIA
 Starting year 2018
 Duration (year-month-day) from 2018-01-01   to  2022-12-31

 Partnership

Take a look of project's partnership.

# participants  country  role  EC contrib. [€] 
1    ACADEMISCH ZIEKENHUIS LEIDEN NL (LEIDEN) coordinator 2˙195˙625.00
2    BATAVIA BIOSCIENCES BV NL (LEIDEN) participant 982˙125.00
3    GENEWERK GMBH DE (HEIDELBERG) participant 596˙250.00
4    MEDIZINISCHE HOCHSCHULE HANNOVER DE (HANNOVER) participant 558˙960.00
5    OSPEDALE SAN RAFFAELE SRL IT (MILANO) participant 532˙500.00
6    INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE FR (PARIS) participant 437˙500.00
7    UNIVERSITY COLLEGE LONDON UK (LONDON) participant 120˙000.00
8    INTERNATIONAL PATIENT ORGANISATION FOR PRIMARY IMMUNODEFICIENCIES UK (TORPOINT) participant 82˙500.00
9    FUNDACIO HOSPITAL UNIVERSITARI VALL D'HEBRON - INSTITUT DE RECERCA ES (BARCELONA) participant 57˙500.00
10    GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST UK (LONDON) participant 57˙500.00
11    LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN DE (MUENCHEN) participant 57˙500.00
12    MEDICAL RESEARCH INFRASTRUCTURE DEVELOPMENT AND HEALTH SERVICES FUND BY THE SHEBA MEDICAL CENTER IL (RAMAT GAN) participant 57˙500.00
13    OSPEDALE PEDIATRICO BAMBINO GESU IT (ROMA) participant 57˙500.00
14    UNIVERSITAET ULM DE (ULM) participant 57˙500.00
15    UNIVERSITY OF NEWCASTLE UPON TYNE UK (NEWCASTLE UPON TYNE) participant 57˙500.00
16    UNIWERSYTET MEDYCZNY IM PIASTOW SLASKICH WE WROCLAWIU PL (WROCLAW) participant 57˙500.00
17    ASSISTANCE PUBLIQUE HOPITAUX DE PARIS FR (PARIS) participant 25˙000.00

Map

 Project 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.

 Deliverables

List of deliverables.
RECOMB Website Websites, patent fillings, videos etc. 2020-04-15 10:43:33

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

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

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