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CureCN

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Adeno Associated Virus Vector Mediated Liver Gene Therapy for Crigler Najjar Syn...

Adeno Associated Virus Vector Mediated Liver Gene Therapy for Crigler Najjar Syndrome 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... ver más

31/12/2023

EIT MANUFACTURING...

6M€

Presupuesto del proyecto: 6M€

Líder del proyecto

EIT MANUFACTURING ASBL No se ha especificado una descripción o un objeto social para esta compañía.

Fecha límite participación Sin fecha límite de participación.

Ver 11 Participantes

Financiación concedida El organismo H2020 notifico la concesión del proyecto el día 2023-12-31

SC1-PM-08-2017: New therapies for rare diseases

I+D

Cerrada hace 8 años

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Información adicional privada

No hay información privada compartida para este proyecto. Habla con el coordinador.

11 Participantes

EIT MANUFACTURING ASBL

4.05M€ | Lider

EURICE EUROPEAN RESEARCH AND...

591.13K€ | Participante

UNINA

147.13K€ | Participante

MHH

90.25K€ | Participante

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Duración del proyecto: 72 meses Fecha Inicio: 2017-12-05
Fecha Fin: 2023-12-31

Líder del proyecto

EIT MANUFACTURING ASBL No se ha especificado una descripción o un objeto social para esta compañía.

Presupuesto del proyecto 6M€

Fecha límite de participación Sin fecha límite de participación.

Descripción del proyecto 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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