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Next-generation AAV vectors for liver-directed gene therapy

Project description

Novel adeno-associated vectors for liver gene transfer

Recombinant adeno-associated vectors (AAV) achieved remarkable success in recent clinical trials for liver gene transfer. However, several hurdles prevent expansion of AAV-mediated therapy to all patients, including transient AAV-mediated expression in proliferating hepatocytes due to episomal AAV genome dilution, hepatotoxicity and immune response against AAVs, and pre-existing immunity to AAV capsids. The EU-funded AAVolution project proposes to find novel small Cas nucleases for in vivo AAV-mediated genome editing with self-replicating episomal AAVs. The aim is to avoid transgene dilution, create synthetic AAVs with reduced toxicity and develop technologies to overcome pre-existing immunity to AAVs. The novel tool set will enable broad expansion of AAV-mediated gene therapy for indications beyond rare diseases.

Coordinator

FONDAZIONE TELETHON ETS
Net EU contribution
€ 1 087 600,00
Address
Via Varese 16/b
00185 Roma
Italy

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Region
Centro (IT) Lazio Roma
Activity type
Research Organisations
Other funding
€ 0,00

Participants (8)

Third-party

Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure.

INNOVAVECTOR SRL
Italy
Net EU contribution
€ 40 000,00
Address
Via Campi Flegrei 34
80078 Pozzuoli

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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
Sud Campania Napoli
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Other funding
€ 0,00
Third-party

Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure.

NEXT GENERATION DIAGNOSTIC SRL
Italy
Net EU contribution
€ 15 000,00
Address
Via Campi Flegrei 34
80078 Pozzuoli

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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
Sud Campania Napoli
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Other funding
€ 0,00
ASSOCIATION GENETHON
France
Net EU contribution
€ 591 000,00
Address
Rue De L Internationale 1 Bis
91002 Evry

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Region
Ile-de-France Ile-de-France Essonne
Activity type
Research Organisations
Other funding
€ 0,00
UNIVERSITA DEGLI STUDI DI TRENTO
Italy
Net EU contribution
€ 551 600,00
Address
Via Calepina 14
38122 Trento

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Region
Nord-Est Provincia Autonoma di Trento Trento
Activity type
Higher or Secondary Education Establishments
Other funding
€ 0,00
FUNDACION PARA LA INVESTIGACION MEDICA APLICADA FIMA
Spain
Net EU contribution
€ 551 600,00
Address
Avenida De Pio Xii 55
31008 Pamplona

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Region
Noreste Comunidad Foral de Navarra Navarra
Activity type
Research Organisations
Other funding
€ 0,00
MEDIZINISCHE HOCHSCHULE HANNOVER
Germany
Net EU contribution
€ 512 200,00
Address
Carl-neuberg-strasse 1
30625 Hannover

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Region
Niedersachsen Hannover Region Hannover
Activity type
Higher or Secondary Education Establishments
Other funding
€ 0,00
CINFERENCE UG
Germany
Net EU contribution
€ 257 000,00
Address
Rheinsberger Strasse 76/77 C/o Fact
10115 Berlin

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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
Berlin Berlin Berlin
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Other funding
€ 0,00
UNIVERSITA DEGLI STUDI DI NAPOLI FEDERICO II
Italy
Net EU contribution
€ 394 000,00
Address
Corso Umberto I, 40
80138 Napoli

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Region
Sud Campania Napoli
Activity type
Higher or Secondary Education Establishments
Other funding
€ 0,00