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EdiGenT

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New Prime Editing and non-viral delivery strategies for Gene Therapy

Genome editing technologies based on CRISPR/Cas systems allow targeted genomic modification with unprecedented precision and have emerged as powerful alternatives to the conventional gene therapy approaches for various human disea... ver más

30/09/2027

SORBONNE UNIVERSIT...

4M€

Presupuesto del proyecto: 4M€

Líder del proyecto

SORBONNE UNIVERSITE 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 7 Participantes

Financiación concedida El organismo HORIZON EUROPE notifico la concesión del proyecto el día 2022-09-13

HORIZON-EIC-2021-PATHFINDERCHALLENGES-01-03: Emerging Technologies in Cell and G... Scope:Cell and gene therapy (CGT) are widely accepted as top biomedical trends for over three years...

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7 Participantes

SORBONNE UNIVERSITE

861.63K€ | Lider

MINISTERE DE L'ENSEIGNEMENT...

735.00K€ | Participante

ZECLINICS

986.25K€ | Participante

ERASMUS MC

727.21K€ | Participante

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Duración del proyecto: 60 meses Fecha Inicio: 2022-09-13
Fecha Fin: 2027-09-30

Líder del proyecto

SORBONNE UNIVERSITE No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Presupuesto del proyecto 4M€

Fecha límite de participación Sin fecha límite de participación.

Descripción del proyecto Genome editing technologies based on CRISPR/Cas systems allow targeted genomic modification with unprecedented precision and have emerged as powerful alternatives to the conventional gene therapy approaches for various human diseases, with a series of clinical trials in progress. However, some crucial challenges remain to be addressed to enhance efficiency and safety and decrease costs of treatments. Current viral-based delivery systems are associated with high risk of toxicity and immunogenicity and remain highly expensive. We will develop a new generation of non-viral delivery systems for gene editing tools based on the use of modified nanoparticles with human-derived protein moieties that will allow targeting the tissue and cells of interest in vivo with minimal adverse effects. Prime editors have raised exciting possibilities for double-strand break free genome editing. However, a major limitation of current prime editors is highly variable efficiency both from one target to another and between cell types. We will design and evaluate novel prime editor tools in order to both increase activity per se and overcome cell-specific limitations.We will test our approach on the hematopoietic system to treat Sickle Cell Disease, avoiding the challenges and risks of hematopoietic stem cell manipulation associated with current gene therapy approaches, and thus providing a treatment much simpler, safer and cost-effective to implement. Our technological breakthroughs address two key obstacles in cell and gene therapy: gene editing efficiency and systemic delivery. The novel prime editors and targeted nanoparticles that we will engineer will be combined to make unprecedented off-the-shelf, recombinant biologics for gene therapy. The versatility of the design of these novel recombinant biologics makes them suitable for the treatment of a vast majority of genetic diseases.

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