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Chemical Rewiring of Oncogenic Transcription

Project description

Rewiring transcription for cancer therapy

Transcription regulation controls when and how genes are expressed, ensuring proper cell function. In cancer, this process is frequently disrupted by mutations, leading to uncontrolled growth and survival. However, directly inhibiting transcription has proven largely ineffective as a therapeutic strategy. The ERC-funded geNEOMORPH project introduces a novel approach based on neomorphic transcriptional compounds (NeoTraCs) that chemically rewire transcriptional regulators. NeoTraCs disrupt oncogenic transcriptional circuits in a targeted, sustained way. Utilising an interdisciplinary strategy, researchers will target key cancer regulators currently lacking effective inhibitors. By establishing a new paradigm for modulating transcription, the project aims to pave the way towards transformative cancer therapies.

Objective

Many cancers are driven by mutations affecting transcription regulation. Through cancer genome sequencing studies, we understand that these aberrations are pervasive through many layers of gene control. Yet, with few exceptions, this knowledge has not precipitated in effective treatments since pharmacologic inhibition of transcription is often infeasible or functionally inconsequential.

To address this limitation, I want to innovate chemical strategies that move beyond loss of function (LOF) approaches. Gene control is organized at the level of proximity. Hence, I hypothesize that transcriptional regulators can be chemically rewired by neomorphic small molecules that recruit functionally opposing gene-regulatory effectors, thereby prompting an acute, deep, and sustained disruption of oncogenic transcriptional circuits. The identification, characterization, and translation of such Neomorphic Transcriptional Compounds (NeoTraCs) will be the subject of the GE|NEomorph proposal. We will focus on NeoTraCs to rewire the repressive PRC2 complex, the p400 chromatin regulator complex, and the non-canonical BAF complex. While these targets have established roles in various cancers, inhibitors are not available or have limited functional impact.

To identify NeoTraCs that address these limitations, we will implement a diverse discovery approach that incorporates hypothesis-driven design with phenotypic strategies geared to co-opt undrugged transcriptional effectors via bivalent NeoTraCs, and unligandable effectors via molecular glue-like NeoTraCs. Identified hits will be characterized via multi-omics approaches. Through careful mechanistic investigation, we will deduce general mechanistic principles, understand their impact on oncogenic transcription and investigate associated translational opportunities.

GE|NEomorph will significantly advance our ability to understand and disrupt transcriptional dysregulation in cancer and pave the way for novel innovative therapies.

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Programme(s)

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Topic(s)

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HORIZON-ERC - HORIZON ERC 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-2024-COG

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Host institution

AITHYRA GMBH RESEARCH INSTITUTE FOR BIOMEDICAL ARTIFICIAL INTELLIGENCE OF THE AUSTRIAN ACADEMY OF SCIENCES
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.

€ 1 999 724,00
Address
HELMUT QUALTINGER GASSE 2 STG 2
1030 Vienna
Austria

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Region
Ostösterreich Wien Wien
Activity type
Private for-profit entities (excluding 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.

€ 1 999 724,00

Beneficiaries (1)

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