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

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.

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)

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INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE

France

EU Contribution

€ 437 500

UNIVERSITY COLLEGE LONDON

United Kingdom

EU Contribution

€ 120 000

GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST

United Kingdom

EU Contribution

€ 57 500

OSPEDALE SAN RAFFAELE SRL

Italy

EU Contribution

€ 532 500

MEDIZINISCHE HOCHSCHULE HANNOVER

Germany

EU Contribution

€ 558 960

UNIVERSITAET ULM

Germany

EU Contribution

€ 57 500

UNIWERSYTET MEDYCZNY IM PIASTOW SLASKICH WE WROCLAWIU

Poland

EU Contribution

€ 57 500

UNIVERSITY OF NEWCASTLE UPON TYNE

United Kingdom

EU Contribution

€ 57 500

INTERNATIONAL PATIENT ORGANISATION FOR PRIMARY IMMUNODEFICIENCIES

United Kingdom

EU Contribution

€ 82 500

BATAVIA BIOSCIENCES BV

Netherlands

EU Contribution

€ 982 125

FUNDACIO HOSPITAL UNIVERSITARI VALL D'HEBRON - INSTITUT DE RECERCA

Spain

EU Contribution

€ 57 500

OSPEDALE PEDIATRICO BAMBINO GESU

Italy

EU Contribution

€ 57 500

ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS

France

EU Contribution

€ 25 000

MEDICAL RESEARCH INFRASTRUCTURE DEVELOPMENT AND HEALTH SERVICES FUND BY THE SHEBA MEDICAL CENTER

Israel

EU Contribution

€ 57 500

LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN

Germany

EU Contribution

€ 57 500

GENEWERK GMBH

Germany

EU Contribution

€ 596 250

Project information

Grant agreement ID: 755170

Status

Ongoing project

  • Start date

    1 January 2018

  • End date

    31 December 2022

Funded under:

H2020-EU.3.1.3.

  • Overall budget:

    € 5 990 460,75

  • EU contribution

    € 5 990 460

Coordinated by:

ACADEMISCH ZIEKENHUIS LEIDEN

Netherlands