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Nanomedicine Approach to Normalize Erythrocyte Maturation in Congenital Anemia by messenger RNA

Objective

Messenger RNA (mRNA) has recently proven itself as a prophylactic modality that can be rapidly developed and employed with high efficacy and low toxicity. Compared to DNA-based gene therapy, mRNA has significant advantages including higher intrinsic safety (non-integrating), lower cost of development, and less stringent regulatory requirements for clinical approval. In the NANEMIAR project, we take advantage of these benefits to kill 2 birds with 1 stone: first, we develop a first-of-its-kind nanomedicine for congenital anemia, a group of rare, inherited blood disorders characterized by ineffective red blood cell production. Our proposed bone marrow-targeted mRNA-based therapy contributes to the first Horizon Europe strategic plan (2021-2024) for new and advanced therapies for non-communicable diseases and is expected to be a vast improvement over current treatment options in terms of efficacy and safety. Second, we provide critical knowledge on targeted non-viral polynucleotide delivery, with a platform that can be exploited for next-generation cell and gene therapy in a broader context. To achieve this, our consortium combines the unique cell-targeting and mRNA knowledge from the Dutch Biotech Mercurna with two excellent research partners (from Spain and France) who have demonstrated expertise in the field of anemia. By focusing on the well-recognized rare disease model, beta-thalassemia, and combining an established mouse model, patient material, and previous drug development experience we build a convincing case to treat congenital anemia.

Fields of science (EuroSciVoc)

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Keywords

Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)

Programme(s)

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Topic(s)

Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.

Funding Scheme

Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.

HORIZON-RIA - HORIZON Research and Innovation Actions

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Call for proposal

Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.

(opens in new window) HORIZON-HLTH-2022-DISEASE-06-two-stage

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Coordinator

FUNDACION PARA LA FORMACION E INVESTIGACION SANITARIAS DE LA REGION DE MURCIA
Net EU contribution

Net EU financial contribution. The sum of money that the participant receives, deducted by the EU contribution to its linked third party. It considers the distribution of the EU financial contribution between direct beneficiaries of the project and other types of participants, like third-party participants.

€ 819 750,00
Address
CALLE CAMPO 12 PABELLON DOCENTE DEL HOSPITAL CLINICO UNIVERSITARIO VIRGEN DE LA ARRIXACA 3 PLANTA EL PALMA
30120 MURCIA
Spain

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Region
Sur Región de Murcia Murcia
Activity type
Research Organisations
Links
Total cost

The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.

€ 907 250,00

Participants (3)

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