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UPGRADE

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Unlocking Precision Gene Therapy

Gene therapy has recently shown remarkable potential to offer definitive treatments for otherwise incurable diseases. Currently, seven gene therapy products have reached the market and many more are entering clinical testing for s... ver más

30/06/2024

FONDAZIONE TELETHO...

15M€

Presupuesto del proyecto: 15M€

Líder del proyecto

FONDAZIONE TELETHON ETS No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Fecha límite participación Sin fecha límite de participación.

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Financiación concedida El organismo H2020 notifico la concesión del proyecto el día 2024-06-30

SC1-BHC-09-2018: Innovation platforms for advanced t... Specific Challenge:Advanced therapies are based on gene, cell or tissue-engineered products which ar...

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12 Participantes

FONDAZIONE TELETHON ETS

4.95M€ | Lider

OSR

N.D. | Participante

NEXT GENERATION DIAGNOSTIC S...

N.D. | Participante

UNITN

1.33M€ | Participante

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Duración del proyecto: 66 meses Fecha Inicio: 2018-12-04
Fecha Fin: 2024-06-30

Líder del proyecto

FONDAZIONE TELETHON ETS No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Presupuesto del proyecto 15M€

Fecha límite de participación Sin fecha límite de participación.

Descripción del proyecto Gene therapy has recently shown remarkable potential to offer definitive treatments for otherwise incurable diseases. Currently, seven gene therapy products have reached the market and many more are entering clinical testing for selected indications. Moreover, emerging technologies for targeted gene editing are complementing the scope of conventional gene transfer, opening the way to precise gene correction and making possible to silence, activate or recode any sequence of interest in the genome. However, in order to realize the full potential of these strategies and broaden application of gene therapy, the field has to solve several major hurdles, including: risk of insertional mutagenesis by gene transfer vectors, limited efficiency and durability of some gene correction strategies, off target effects of editing tools, poor tissue targeting and immune response to editing components and delivery vehicles. UPGRADE will offer radical new solutions to overcome these hurdles. We will exploit and further develop disruptive new technologies for precision gene and epigenome editing and for site-specific transgene insertion, and stringently characterize their specificity and cellular responses. We will combine these improved technologies with advanced viral and non-viral vectors enabling cell/tissue targeting and immune evasion, to generate prototypes of advanced medicinal products (AMP). The safety and efficacy profile of each AMP will be stringently validated in tissues (hematopoiesis, heart and skeletal muscle, liver, retina) and disease models (muscle wasting, storage and blood disorders, hypercholesterolemia) paradigmatic for unmet medical need and potential long-term cure, if the limitations of current gene therapy strategies are overcome. These AMPs represent versatile products portable to the treatment of several other diseases because of related pathogenesis or correction strategies, thus providing the basis for tackling diseases affecting large patient groups.

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