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RECOMB SIGNED

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

Total Cost €

EC-Contrib. €

Partnership

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Outcomes and
results

RECOMB project word cloud

Explore the words cloud of the RECOMB project. It provides you a very rough idea of what is the project "RECOMB" about.

fill therapeutic gmp underlying gene gt unmet trials safety deficient genes advocacy recomb activating input extension autologous clinical scid medical leader therapy forms occurred treatment expression molecular ema performing defects regulatory rare conduct patients groups disorders logical successful 70 hematopoietic recombination group lentiviral patient batches complexities rag orphan category global functional integration multicenter stem stage inherited readouts ada phenotypic made diseases cell sites assays successfully immune types multinational

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 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.	NL (LEIDEN)	coordinator	2˙195˙625.00
2	BATAVIA BIOSCIENCES BV BATAVIA BIOSCIENCES BV Organization address address: ZERNIKEDREEF 16 city: LEIDEN postcode: 2333 CL website: www.bataviabioservices.com contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	NL (LEIDEN)	participant	982˙125.00
3	GENEWERK GMBH GENEWERK GMBH Organization address address: IM NEUENHEIMER FELD 582 city: HEIDELBERG postcode: 69120 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.	DE (HEIDELBERG)	participant	596˙250.00
4	MEDIZINISCHE HOCHSCHULE HANNOVER MEDIZINISCHE HOCHSCHULE HANNOVER Organization address address: Carl-Neuberg-Strasse 1 city: HANNOVER postcode: 30625 website: www.mh-hannover.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 (HANNOVER)	participant	558˙960.00
5	OSPEDALE SAN RAFFAELE SRL OSPEDALE SAN RAFFAELE SRL Organization address address: VIA OLGETTINA 60 city: MILANO postcode: 20132 website: www.hsr.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 (MILANO)	participant	532˙500.00
6	INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE Organization address address: RUE DE TOLBIAC 101 city: PARIS postcode: 75654 website: www.inserm.fr contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	FR (PARIS)	participant	437˙500.00
7	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)	participant	120˙000.00
8	INTERNATIONAL PATIENT ORGANISATION FOR PRIMARY IMMUNODEFICIENCIES INTERNATIONAL PATIENT ORGANISATION FOR PRIMARY IMMUNODEFICIENCIES Organization address address: MAIN ROAD FIRSIDE city: TORPOINT postcode: PL11 3LE 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 (TORPOINT)	participant	82˙500.00
9	FUNDACIO HOSPITAL UNIVERSITARI VALL D'HEBRON - INSTITUT DE RECERCA FUNDACIO HOSPITAL UNIVERSITARI VALL D'HEBRON - INSTITUT DE RECERCA Organization address address: PASSEIG VALL D HEBRON 119-129 EDIFICIO DE RECERCA city: BARCELONA postcode: 8035 website: http://www.vhir.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 (BARCELONA)	participant	57˙500.00
10	GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST Organization address address: GREAT ORMOND STREET city: LONDON postcode: WC1N 3JH 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)	participant	57˙500.00
11	LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN Organization address address: GESCHWISTER SCHOLL PLATZ 1 city: MUENCHEN postcode: 80539 website: www.uni-muenchen.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 (MUENCHEN)	participant	57˙500.00
12	MEDICAL RESEARCH INFRASTRUCTURE DEVELOPMENT AND HEALTH SERVICES FUND BY THE SHEBA MEDICAL CENTER MEDICAL RESEARCH INFRASTRUCTURE DEVELOPMENT AND HEALTH SERVICES FUND BY THE SHEBA MEDICAL CENTER Organization address address: TEL HASHOMER SHEBA MEDICAL CENTER city: RAMAT GAN postcode: 52621 website: http://www.sheba.co.il contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	IL (RAMAT GAN)	participant	57˙500.00
13	OSPEDALE PEDIATRICO BAMBINO GESU OSPEDALE PEDIATRICO BAMBINO GESU Organization address address: PIAZZA SANT ONOFRIO 4 city: ROMA postcode: 165 website: www.ospedalebambinogesu.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	57˙500.00
14	UNIVERSITAET ULM UNIVERSITAET ULM Organization address address: HELMHOLTZSTRASSE 16 city: ULM postcode: 89081 website: www.uni-ulm.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 (ULM)	participant	57˙500.00
15	UNIVERSITY OF NEWCASTLE UPON TYNE UNIVERSITY OF NEWCASTLE UPON TYNE Organization address address: KINGS GATE city: NEWCASTLE UPON TYNE postcode: NE1 7RU website: http://www.ncl.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 (NEWCASTLE UPON TYNE)	participant	57˙500.00
16	UNIWERSYTET MEDYCZNY IM PIASTOW SLASKICH WE WROCLAWIU UNIWERSYTET MEDYCZNY IM PIASTOW SLASKICH WE WROCLAWIU Organization address address: UL PASTEURA 1 city: WROCLAW postcode: 50 367 website: www.am.wroc.pl contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	PL (WROCLAW)	participant	57˙500.00
17	ASSISTANCE PUBLIQUE HOPITAUX DE PARIS ASSISTANCE PUBLIQUE HOPITAUX DE PARIS Organization address address: 3 AVENUE VICTORIA city: PARIS postcode: 75000 website: http://www.aphp.fr contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	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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