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Adeno-Associated Virus Vector-Mediated Liver Gene Therapy for Crigler-Najjar Syndrome

Adeno-Associated Virus Vector-Mediated Liver Gene Therapy for Crigler-Najjar Syndrome

Objective

Crigler-Najjar syndrome (CN) is a rare recessive disorder caused by mutations in the uridine diphosphate glucuronosyltransferase 1A1(UGT1A1) gene. CN is a life-threatening disease with no cure which constitutes a severe burden for the patients, their families, and the society. CureCN has the objective of developing a curative gene therapy for CN syndrome based on liver gene transfer with and adeno-associated virus (AAV) vector expressing the UGT1A1 transgene. Additional goals of CureCN are to develop strategies to allow for vector re-administration and to address the issue of pre-existing anti-AAV neutralizing antibodies (NAbs), which prevent large proportion of seropositive patients from receiving AAV mediated gene therapy. Proof-of-concept studies of AAV8-UGT1A1 gene transfer provide a strong rationale for the safety and efficacy of gene therapy for CN. CureCN proposes to carry out an open-label, multicenter clinical trial of AAV8-UGT1A1 gene transfer to prove the safety and efficacy of the therapy in severe CN patients, and file for marketing authorization in Europe at the end of the study. CureCN will also produce enabling data for the clinical translation of a groundbreaking immunomodulatory strategy to allow for vector administration. Additionally, a technology for the selective removal of anti-AAV NAbs from the bloodstream of seropositive patients will be developed. The goal of these studies is to ultimately allow all CN patients to access AAV8-UGT1A1 gene therapy. CureCN is a patient-driven initiative that gathers top clinicians and scientists; it also includes small medium enterprises in its partners, to foster economic growth and valorization of intellectual property. CureCN sets itself in the ambitious goal set by the IRDiRC by 2020 by developing a curative treatment for CN syndrome. Importantly, it validates technologies that will broaden the scope of gene therapy, thus will have an impact on the development of treatments for several other rare diseases.
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Coordinator

ASSOCIATION GENETHON

Address

Rue De L Internationale 1 Bis
91002 Evry

France

Activity type

Higher or Secondary Education Establishments

EU Contribution

€ 3 738 478,75

Participants (10)

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ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS

France

EU Contribution

€ 172 083,75

Academisch Medisch Centrum bij de Universiteit van Amsterdam

Netherlands

EU Contribution

€ 526 718,75

MEDIZINISCHE HOCHSCHULE HANNOVER

Germany

EU Contribution

€ 149 000

AZIENDA OSPEDALIERA PAPA GIOVANNI XXIII

Italy

EU Contribution

€ 147 500

UNIVERSITA DEGLI STUDI DI NAPOLI FEDERICO II

Italy

EU Contribution

€ 147 125

GENOSAFE SAS

France

EU Contribution

€ 207 017,50

ASSOCIATION FRANCAISE DE CRIGLER NAJJAR

France

EU Contribution

€ 92 000

MC TOXICOLOGY CONSULTING GMBH

Austria

EU Contribution

€ 156 872,50

UNIVERSITY OF LEICESTER

United Kingdom

EU Contribution

€ 396 182,50

EURICE EUROPEAN RESEARCH AND PROJECT OFFICE GMBH

Germany

EU Contribution

€ 516 125

Project information

Grant agreement ID: 755225

Status

Ongoing project

  • Start date

    1 January 2018

  • End date

    31 December 2022

Funded under:

H2020-EU.3.1.3.

  • Overall budget:

    € 6 249 103,75

  • EU contribution

    € 6 249 103,75

Coordinated by:

ASSOCIATION GENETHON

France