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Kits for advanced polymer-lipid nanocarriers for targeted delivery of RNAs to cardiac and skeletal muscle cells

Project description

Advancing delivery of RNA therapeutics to muscle tissue

RNA therapeutics harness the power of RNA molecules, such as small interfering RNAs (siRNAs) and messenger RNAs (mRNAs), to target and modulate specific genes. Despite their great potential, their utilisation has been limited to the treatment of liver diseases and COVID-19 vaccines mainly due to the lack of efficient delivery vehicles. Funded by the European Research Council, the POLIRNA project aims to develop non-toxic lipid-based nanocarriers formulated for the efficient delivery of RNA therapeutics to cardiomyocytes and skeletal muscle cells. Collaboration with experts and industry support will facilitate technology validation and potentially lead to a start-up company and investment opportunities.

Objective

Currently, RNA therapies are available on the clinics to treat liver diseases and as COVID-19 vaccines. Hence, their therapeutic potential still remains largely unexploited for the treatment of other disorders, such as age-related, rare genetic and infectious diseases. One key problem for effective preclinical research on new RNA therapies is the lack of safe, efficient and cell-specific delivery vehicles. Gold-standard cationic lipid transfection reagents suffer from dose-dependent cytotoxicity, inability for cell-targeted delivery and poor stability. Clinically approved lipid nanoparticles efficiently target liver cells, while attempts to target other cell types have achieved limited success. In POLIRNA I will develop and validate a new safe, efficient, and versatile platform for RNA release to target multiple cell types, based on ligand-functionalized polymer-lipid nanocarriers, by exploiting a new patented method from my ERC-funded project BIORECAR (772168). POLIRNA consists of “kits for research use”, allowing user-friendly preparation of nanocarriers able to effectively deliver non-coding RNAs (such as siRNAs and microRNAs) to target cardiomyocytes and skeletal muscle cells for preclinical research studies on cardiac regenerative medicine and muscle-related diseases (e.g. neuromuscular diseases). Technology development and validation will benefit from collaboration with major experts in the fields and companies supporting the innovation route from lab-to-market. My previous experience as coordinator of research and innovation projects and as co-founding member of an academic spin-off company, and the current involvement of my team in business plan competitions will drive the process of technology validation, with the aim to strengthen technology value proposition versus current gold-standard solutions, found a start-up company and attract investments.

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Programme(s)

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Topic(s)

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Funding Scheme

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HORIZON-ERC-POC - HORIZON ERC Proof of Concept Grants

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Call for proposal

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(opens in new window) ERC-2022-POC2

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Host institution

POLITECNICO DI TORINO
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.

€ 150 000,00
Address
CORSO DUCA DEGLI ABRUZZI 24
10129 Torino
Italy

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Region
Nord-Ovest Piemonte Torino
Activity type
Higher or Secondary Education Establishments
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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.

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Beneficiaries (1)

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