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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.

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