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CureCN SIGNED

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

Total Cost €

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EC-Contrib. €

0

Partnership

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 CureCN project word cloud

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

immunomodulatory    adeno    ugt1a1    disease    threatening    uridine    data    mediated    caused    multicenter    proof    life    recessive    marketing    valorization    diphosphate    enterprises    seropositive    removal    virus    trial    clinicians    broaden    society    treatment    diseases    families    intellectual    strategy    proposes    curative    irdirc    file    cure    authorization    glucuronosyltransferase    transfer    safety    liver    treatments    2020    scientists    issue    medium    groundbreaking    aav    patient    itself    patients    ultimately    bloodstream    rare    syndrome    constitutes    carry    prevent    economic    property    mutations    selective    additional    scope    vector    validates    aav8    transgene    small    cn    administration    goals    translation    strategies    disorder    additionally    nabs    curecn    expressing    antibodies    burden    therapy    proportion    re    efficacy    prove    neutralizing    clinical    anti    rationale    technologies    1a1    najjar    receiving    severe    gene    crigler    label   

Project "CureCN" data sheet

The following table provides information about the project.

Coordinator
ASSOCIATION GENETHON 

Organization address
address: RUE DE L INTERNATIONALE 1 BIS
city: EVRY
postcode: 91002
website: www.genethon.fr

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 France [FR]
 Project website https://curecn.eu/
 Total cost 6˙249˙103 €
 EC max contribution 6˙249˙103 € (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    ASSOCIATION GENETHON FR (EVRY) coordinator 3˙738˙478.00
2    ACADEMISCH MEDISCH CENTRUM BIJ DE UNIVERSITEIT VAN AMSTERDAM NL (AMSTERDAM) participant 526˙718.00
3    EURICE EUROPEAN RESEARCH AND PROJECT OFFICE GMBH DE (ST INGBERT) participant 516˙125.00
4    UNIVERSITY OF LEICESTER UK (LEICESTER) participant 396˙182.00
5    GENOSAFE SAS FR (EVRY) participant 207˙017.00
6    ASSISTANCE PUBLIQUE HOPITAUX DE PARIS FR (PARIS) participant 172˙083.00
7    MC TOXICOLOGY CONSULTING GMBH AT (WIEN) participant 156˙872.00
8    MEDIZINISCHE HOCHSCHULE HANNOVER DE (HANNOVER) participant 149˙000.00
9    AZIENDA OSPEDALIERA PAPA GIOVANNI XXIII IT (BERGAMO) participant 147˙500.00
10    UNIVERSITA DEGLI STUDI DI NAPOLI FEDERICO II IT (NAPOLI) participant 147˙125.00
11    ASSOCIATION FRANCAISE DE CRIGLER NAJJAR FR (CLAMART) participant 92˙000.00

Map

 Project 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.

 Deliverables

List of deliverables.
Public project website Websites, patent fillings, videos etc. 2020-02-18 10:25:17

Take a look to the deliverables list in detail:  detailed list of CureCN deliverables.

 Publications

year authors and title journal last update
List of publications.
2019 Fanny Collaud, Giulia Bortolussi, Laurence Guianvarc’h, Sem J. Aronson, Thierry Bordet, Philippe Veron, Severine Charles, Patrice Vidal, Marcelo Simon Sola, Stephanie Rundwasser, Delphine G. Dufour, Florence Lacoste, Cyril Luc, Laetitia v. Wittenberghe, Samia Martin, Christine Le Bec, Piter J. Bosma, Andres F. Muro, Giuseppe Ronzitti, Matthias Hebben, Federico Mingozzi
Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome
published pages: 157-174, ISSN: 2329-0501, DOI: 10.1016/j.omtm.2018.12.011
Molecular Therapy - Methods & Clinical Development 12 2020-02-18

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The information about "CURECN" are provided by the European Opendata Portal: CORDIS opendata.

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