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ARDAT

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Cerrado

Accelerating Research Development for Advanced Therapies

Major current hurdles for wide clinical use of AAV vectors are attributable primarily to: (i) host elimination by both immune and non-immune sequestering mechanisms – such neutralization by host antibody responses critically limit... ver más

31/10/2025

USFD

25M€

Presupuesto del proyecto: 25M€

Líder del proyecto

THE UNIVERSITY OF SHEFFIELD No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Fecha límite participación Sin fecha límite de participación.

Ver 33 Participantes

Financiación concedida El organismo H2020 notifico la concesión del proyecto el día 2020-11-11

IMI2-2019-18-05: Accelerating research & innovation... Specific Challenge:Curative or near curative therapies for rare and orphan diseases have been a long...

I+D

Cerrada hace 5 años

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Características del participante

Este proyecto no cuenta con búsquedas de partenariado abiertas en este momento.

Información adicional privada

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

33 Participantes

USFD

2.70M€ | Lider

UNIVERSITY OF EASTERN FINLAN...

571.25K€ | Participante

IBET

400.00K€ | Participante

JANSSEN PHARMACEUTICA NV

1.10M€ | Participante

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Duración del proyecto: 59 meses Fecha Inicio: 2020-11-11
Fecha Fin: 2025-10-31

Líder del proyecto

THE UNIVERSITY OF SHEFFIELD No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Presupuesto del proyecto 25M€

Fecha límite de participación Sin fecha límite de participación.

Descripción del proyecto Major current hurdles for wide clinical use of AAV vectors are attributable primarily to: (i) host elimination by both immune and non-immune sequestering mechanisms – such neutralization by host antibody responses critically limits the possibility of repeated AAV delivery; (ii) AAVs are prevalent in the environment and hence a large proportion of the population carry AAV antibodies (up to 80%)– this pre-existing immunity renders AAV unable to infect target cells forcing substantial patient cohorts to be excluded from clinical trials. The current proposal is founded on compelling track record in the field and brings together a ‘best-with-best’ multidisciplinary team of international leading academic and EFPIA partners with complimentary expertise in gene therapy, immunology, chemistry, engineering, biotechnology, drug safety, viral vector production, regulatory and clinical trials. The overall goal is to analyse the currently available clinical data and then design preclinical and clinical studies to fill the knowledge gaps in advanced therapies development. Our main aims are to: 1) Develop improved model systems for predicting product immunogenicity in humans. This will be achieved by generating human and NHP 3D hepatic models; 2) Enhance our understanding of gene/cell therapy drug metabolism inside a host of cell types. The plan is to define metabolism of the therapeutic vector genome in different cell types to understand whether rates of degradation, episomal maintenance, or integration, and metabolic stress induced by AAV vector transgene expression vary from cell to cell. We will then adopt strategies to mitigate the loss of vector genomes and improve persistence; 3) Use diverse clinical expertise to establish the clinical factors around pre-existing immunity limiting patient access to advanced therapies therapy; 4) Engage regulators to ensure that the concepts and the data generated through this IMI programme will fill the gaps and support furture trials.

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