Summary
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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| Web resources: | https://cordis.europa.eu/project/id/101113522 |
| Start date: | 01-10-2023 |
| End date: | 31-03-2025 |
| Total budget - Public funding: | - 150 000,00 Euro |
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Original description
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.Status
SIGNEDCall topic
ERC-2022-POC2Update Date
31-07-2023
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