Objective
Genetic disorders cause substantial childhood morbidity and mortality. They affect more than 30 million individuals in Europe alone and are estimated to cost over $1 trillion each year. Therapeutic options remain limited.
Prime editing is an emerging precise gene-editing approach that promises causal treatment of genetic disorders by correcting the disease-causing genetic variants. Whereas conventional gene therapies add or knock out a gene, prime editing can rewrite endogenous DNA with high precision. However, the editing efficiency is often too low to anticipate clinical effects, especially for mutations in AT-rich DNA loci, where interaction between the prime editor and DNA is weaker. Consequently, (pre)clinical programs are biased toward GC-rich targets, constraining prime editing’s impact to minimal clinical breakthroughs (to date, only one clinical trial).
As part of my 2022 ERC Starting Grant PRIME, my team developed AT-PRIME to overcome this bias by bypassing the AT-rich DNA limitation. Preliminary data shows a 7x increase in editing efficiency in an AT-rich locus. Based on these results, we predict that up to 77% of pathogenic variants can be corrected using AT-PRIME, compared to only 17% with state-of-the-art prime editors. This would revolutionize prospects for currently untreatable patients, industry and academic research.
I now aim to explore the technical and commercial feasibility of this innovation. My team will generate essential proof-of-concept editing efficiency data in commercially relevant, difficult-to-edit mutations, while exploring strategies for scalable Good Manufacturing Practices production. In parallel, we will consolidate and strengthen our IP-position, execute a comprehensive freedom-to-operate strategy, and analyse the market landscape. Based on the technical work and the identified market needs, we will develop a solid business strategy. As such, this project will develop the most promising commercialization routes.
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.
- social sciences sociology demography mortality
- natural sciences biological sciences genetics DNA
- natural sciences biological sciences genetics mutation
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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)
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
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.
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HORIZON.1.1 - European Research Council (ERC)
MAIN PROGRAMME
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Topic(s)
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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
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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-2026-POC
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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.
3584 CX Utrecht
Netherlands
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.