Project description
Innovative particle for cell-specific delivery
Cell-specific cargo delivery is a critical issue in the field of RNA-based therapeutics. The delivery relies on lipid nanoparticles which encapsulate RNA, but this method lacks specificity. Viral vectors are currently the only tissue-specific cargo delivery option. Nevertheless, viral systems also have disadvantages, such as oncogenicity and pre-existing immunity to the viral carrier. The nanoVAST particle is a patented vesicular phospholipid bilayer densely decorated with a single protein that can be fused to a targeting molecule of interest through specific, efficient and separately patented chemistry. The EU-funded nanoVAST project proposes to use the nanoVAST particle to deliver specific RNA cargo to CD19+ B cells and functionally alter the targeted cells.
Objective
Cell-specific cargo delivery is a key remaining problem in the field of RNA-based therapeutics. Thus far, delivery has relied on lipid nanoparticles (LNPs) which encapsulate RNA with high efficiency, but broadly lack in specificity. Viral vectors are currently the only FDA approved tissue-specific cargo delivery option, with specificity being the result of manipulation of outer capsid proteins. However, viral systems are also associated with drawbacks such as oncogenicity, antigenicity and pre-existing immunity to the viral carrier itself. The ideal carrier system would involve LNP-efficient cargo encapsulation together with specific targeting in the absence of oncogenicity or immunogenicity.
DKFZ and Panosome GmbH have together developed this exact particle – the nanoVAST: a patented vesicular phospholipid bilayer densely decorated with a single protein that can be fused to a targeting molecule of interest through specific, efficient, and separately patented chemistry. Importantly, the attachment of the targeting component relies on a coupling of the vesicle to the targeting moiety, rather than a genetic manipulation of the carrier itself (as is the case with viral vectors), giving this system an unparalleled level of versatility. Additionally, our vesicular system is inherently fusogenic with target membranes, thus permitting cargo delivery directly to the cytoplasm and avoiding the reliance on the incredibly inefficient “endosomal escape” mechanism that plagues LNPs (it is estimated that conventional LNPs only deliver approximately 1% or less of their payload into the cytoplasm).
This ERC PoC proposes to use the nanoVAST particle to (a) deliver specific RNA cargo to CD19+ B cells; (b) transport within the cells this cargo to the endogenous RNA editing machinery and (c) functionally alter the surface of the targeted cells. Through the PoC we aim to accelerate nanoVAST, our precision payload delivery system, towards direct clinical applications.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
- natural sciences biological sciences biochemistry biomolecules proteins
- natural sciences biological sciences genetics RNA
- engineering and technology nanotechnology nano-materials
- natural sciences biological sciences genetics genomes
You need to log in or register to use this function
We are sorry... an unexpected error occurred during execution.
You need to be authenticated. Your session might have expired.
Thank you for your feedback. You will soon receive an email to confirm the submission. If you have selected to be notified about the reporting status, you will also be contacted when the reporting status will change.
Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
-
HORIZON.1.1 - European Research Council (ERC)
MAIN PROGRAMME
See all projects funded under this programme
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.
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.
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-ERC-POC - HORIZON ERC Proof of Concept Grants
See all projects funded under this funding scheme
Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
(opens in new window) ERC-2022-POC2
See all projects funded under this callHost institution
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.
69120 Heidelberg
Germany
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.