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Transcription Factor Gene Therapy for Bradyarrhythmias

Project description

Gene-therapy assisted heart pacemaker

Bradyarrhythmia is a disorder characterised by a slow heart rate and an abnormality in the electrical signals that control the heart's natural pacemaker. This can lead to symptoms such as fatigue and fainting but can be life-threatening in some cases. Funded by the European Innovation Council, the TRACTION project introduces BradyTx-01, an innovative gene therapy approach, specifically designed to reprogramme heart muscle cells into pacemaker cells. The product employs adeno-associated viral vector technology to control transcription factor overexpression. The consortium will validate this new strategy in preclinical studies before proceeding with further clinical and commercial development. This biological pacemaker can improve cardiac function and potentially last a lifetime, making it an attractive alternative to traditional pacemakers.

Objective

PacingCure has developed BradyTx-01: a novel gene therapy medicinal product (GTMP) to cure life-threatening cardiac pacing dysfunctions by reprogramming cardiomyocytes into pacemaker cells. BradyTx-01 is based on a novel method to optimally control transcription factor overexpression through adeno-associated Virus (AAV) mediated gene transfer. The biological pacemaker that is created (1) improves cardiac function, (2) responds to metabolic demand, (3) can be delivered using minimally invasive techniques and (4) potentially lasts a lifetime.

In the TRACTION project, PacingCure together with AUMC will further optimize AAV-mediated transcription vector gene therapy for pacemaker applications, validating the novel AAV vector technology in mice, pigs and non-human primates. Subsequently, a large-scale manufacturing process will be designed to release the materials that will be used for validating and demonstrating long-term safety and efficacy in porcine studies, thereby establishing preclinical proof-of-concept of BradyTx-01.

In parallel, we will validate and optimize the technology-product-market fit of BradyTx-01, and the evolving value propositions will be tested and optimized with relevant stakeholders. We will define the optimal regulatory strategies, perform health technology assessments and develop the optimal business strategy, thereby setting the stage for effective clinical and commercial development of BradyTx-01.

Within the TRACTION project, we will take the next steps in our development, while also establishing the optimal strategy for our trajectory towards the clinical stage and commercialization of BradyTx-01. The TRACTION project will thus facilitate the complete transition of PacingCure into a fully operational company with an attractive and investment-ready business proposition.

Keywords

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Programme(s)

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Topic(s)

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Funding Scheme

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HORIZON-EIC - HORIZON EIC Grants

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Call for proposal

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(opens in new window) HORIZON-EIC-2022-TRANSITION-01

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Coordinator

PACINGCURE B.V.
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.

€ 2 049 687,00
Address
ROETERSSTRAAT 35
1018 WB Amsterdam
Netherlands

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SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
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.

€ 2 499 968,75

Participants (1)

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