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DevelopIng Genetic medicines for Severe Combined Immunodeficiency (SCID)

Objective

Severe combined immunodeficiency (SCID) is a devastating rare disorder of immune system development. Affected infants are born without functional immune systems and die within the first year of life unless effective treatment is given. Treatment options are limited to allogeneic haematopoietic stem cell transplantation and autologous stem cell gene therapy. Over the last 15 years, gene therapy for two forms of SCID (SCID-X1 and ADA SCID) has shown significant safety and efficacy in correcting the immunodeficiency and allowing children to live normal lives. Proof of concept of gene therapy for 3 other SCID forms has also been shown by members of the proposed SCIDNET consortium and is ready for translation into clinical trials. We are therefore in a position whereby, over the next 4 years, we can offer gene therapy as a curative option for over 80% of all forms of SCID in Europe. Importantly for 1 of these conditions (ADA SCID) we will undertake clinical trials that will lead to marketing authorisation of the gene therapy product as a licensed medicine. In addition, we will investigate the future technologies that will improve the safety and efficacy of gene therapy for SCID.

Our proposal addresses an unmet clinical need in SCID, which is classified as a rare disease according to EU criteria (EC regulation No. 141/2000). The proposal also addresses the need to develop an innovative treatment such as gene therapy from early clinical trials though to a licensed medicinal product through involvement with regulatory agencies and is in keeping with the ambitions of the IRDiRC. The lead ADA SCID programme has Orphan Drug Designation and clinical trial design is assisted by engagement with the European medicines Agency. The ADA SCID trial will act as a paradigm for the development of the technologies and processes that will allow gene therapy for not only SCID, but also other bone marrow disorders, to become authorised genetic medicines in the future.

Coordinator

UNIVERSITY COLLEGE LONDON

Address

Gower Street
Wc1e 6bt London

United Kingdom

Activity type

Higher or Secondary Education Establishments

EU Contribution

€ 1 549 174

Participants (11)

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ACADEMISCH ZIEKENHUIS LEIDEN

Netherlands

EU Contribution

€ 799 877

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE

France

EU Contribution

€ 850 002

GENEWERK GMBH

Germany

EU Contribution

€ 699 750

OSPEDALE SAN RAFFAELE SRL

Italy

EU Contribution

€ 474 250

UNIVERSITAETSKLINIKUM FREIBURG

Germany

EU Contribution

€ 499 300

ASSOCIATION GENETHON

France

EU Contribution

€ 800 000

MEDIZINISCHE HOCHSCHULE HANNOVER

Germany

EU Contribution

€ 509 562

GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST

United Kingdom

EU Contribution

€ 423 198

MILTENYI BIOTEC GMBH

Germany

EU Contribution

€ 296 200

CENTRE HOSPITALIER UNIVERSITAIRE VAUDOIS

Switzerland

INTERNATIONAL PATIENT ORGANISATION FOR PRIMARY IMMUNODEFICIENCIES

United Kingdom

EU Contribution

€ 25 000

Project information

Grant agreement ID: 666908

Status

Ongoing project

  • Start date

    1 January 2016

  • End date

    31 December 2019

Funded under:

H2020-EU.3.1.3.

  • Overall budget:

    € 7 474 316

  • EU contribution

    € 6 926 313

Coordinated by:

UNIVERSITY COLLEGE LONDON

United Kingdom