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

Objective

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.

Coordinator

SORBONNE UNIVERSITE
Net EU contribution
€ 816 875,00
Address
21 Rue De L'ecole De Medecine
75006 Paris
France

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Region
Ile-de-France Ile-de-France Paris
Activity type
Higher or Secondary Education Establishments
Other funding
€ 816 875,00

Participants (4)

ERASMUS UNIVERSITAIR MEDISCH CENTRUM ROTTERDAM
Netherlands
Net EU contribution
€ 727 210,00
Address
Dr Molewaterplein 40
3015 GD Rotterdam

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Region
West-Nederland Zuid-Holland Groot-Rijnmond
Activity type
Higher or Secondary Education Establishments
Other funding
€ 727 210,00
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
Net EU contribution
€ 647 500,00
Address
Rue De Tolbiac 101
75654 Paris

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Region
Ile-de-France Ile-de-France Paris
Activity type
Research Organisations
Other funding
€ 647 500,00
ZECLINICS SL
Spain
Net EU contribution
€ 986 250,00
Address
Ctra De Can Ruti, Cami De Les Escoles, S/n Laboratori P0-8, Edifici Igtp Muntanya
08916 Barcelona

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
Este Cataluña Barcelona
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Other funding
€ 986 250,00
IMAGINE INSTITUT DES MALADIES GENETIQUES NECKER ENFANTS MALADES FONDATION
France
Net EU contribution
€ 735 000,00
Address
24 Bd Du Montparnasse
75015 Paris 15

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Region
Ile-de-France Ile-de-France Paris
Activity type
Research Organisations
Other funding
€ 735 000,00