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NoStop: Personalised Small Molecule Gene Therapy

Objective

Premature termination codons (PTCs) account for 10–20% of genetic disease cases and represent a major mechanism of tumor suppressor gene inactivation. A therapeutic approach capable of reverting PTCs into sense codons would have broad applicability, benefiting millions of patients across diverse genetic disorders and cancers. Readthrough therapy offers this potential; however, its clinical impact has so far been limited by disappointing trial outcomes.
Using a combination of in-house–developed high-throughput human readthrough assays and machine learning, we recently demonstrated that a key factor underlying this limited success is the strong sequence specificity of readthrough drugs, which varies markedly among compounds. With the NoStop platform, our goal is to build on this validated technology to transform the readthrough therapy field by systematically generating comprehensive PTC-specificity maps for all known readthrough compounds, while simultaneously establishing a sequence-tailored high-throughput drug discovery pipeline to identify novel proprietary readthrough drugs. If successful, NoStop will generate invaluable data to enable the transition of readthrough therapy from a one-size-fits-all approach to a personalized medicine paradigm.

Fields of science (EuroSciVoc)

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Programme(s)

Multi-annual funding programmes that define the EU’s priorities for research and innovation.

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-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.

(opens in new window) ERC-2025-POC

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Host institution

FUNDACIO CENTRE DE REGULACIO GENOMICA
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.

€ 107 000,00
Address
CARRER DOCTOR AIGUADER 88
08003 Barcelona
Spain

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Region
Este Cataluña Barcelona
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.

No data

Beneficiaries (2)