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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.

Field of science

  • /medical and health sciences/medical biotechnology/cells technologies/stem cells
  • /medical and health sciences/basic medicine/pharmacology and pharmacy/pharmaceutical drug
  • /medical and health sciences/clinical medicine/transplantation
  • /medical and health sciences/medical biotechnology/genetic engineering/gene therapy
  • /medical and health sciences/basic medicine/immunology

Call for proposal

H2020-PHC-2015-two-stage
See other projects for this call

Funding Scheme

RIA - Research and Innovation action

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)

ACADEMISCH ZIEKENHUIS LEIDEN
Netherlands
EU contribution
€ 799 877
Address
Albinusdreef 2
2333 ZA Leiden
Activity type
Higher or Secondary Education Establishments
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
EU contribution
€ 850 002
Address
Rue De Tolbiac 101
75654 Paris
Activity type
Research Organisations
GENEWERK GMBH
Germany
EU contribution
€ 699 750
Address
Im Neuenheimer Feld 582
69120 Heidelberg
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
OSPEDALE SAN RAFFAELE SRL
Italy
EU contribution
€ 474 250
Address
Via Olgettina 60
20132 Milano
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
UNIVERSITAETSKLINIKUM FREIBURG
Germany
EU contribution
€ 499 300
Address
Hugstetter Strasse 49
79106 Freiburg
Activity type
Higher or Secondary Education Establishments
ASSOCIATION GENETHON
France
EU contribution
€ 800 000
Address
Rue De L Internationale 1 Bis
91002 Evry
Activity type
Research Organisations
MEDIZINISCHE HOCHSCHULE HANNOVER
Germany
EU contribution
€ 509 562
Address
Carl-neuberg-strasse 1
30625 Hannover
Activity type
Higher or Secondary Education Establishments
GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST
United Kingdom
EU contribution
€ 423 198
Address
Great Ormond Street
WC1N 3JH London
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
MILTENYI BIOTEC GMBH
Germany
EU contribution
€ 296 200
Address
Friedrich Ebert Strasse 68
51429 Bergish Gladbach
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
CENTRE HOSPITALIER UNIVERSITAIRE VAUDOIS
Switzerland
EU contribution
€ 0
Address
Rue Du Bugnon 21
1011 Lausanne
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
INTERNATIONAL PATIENT ORGANISATION FOR PRIMARY IMMUNODEFICIENCIES
United Kingdom
EU contribution
€ 25 000
Address
Main Road Firside
PL11 3LE Torpoint
Activity type
Other