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GEREMY

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Gene Therapy for treatment of rare inherited Arrhythmogenic Cardiomyopathy

Development of a cure for rare cardiac diseases is a risky, costly and time-consuming process, which is hampered by insufficient insight into pathophysiological mechanisms of the diseases and lack of relevant disease models. The G... see more

30/04/2027

STICHTING NETHERLA...

7M€

Project Budget: 7M€

Project leader

STICHTING NETHERLANDS HEART INSTITUTE No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

PARTICIPATION DEPralty Sin fecha límite de participación.

Financing granted El organismo HORIZON EUROPE notifico la concesión del proyecto The day 2023-05-01

HORIZON-HLTH-2022-DISEASE-06-02-two-stage: Pre-clinical development of the nex... Expected Outcome:This topic aims at supporting activities that are enabling or contributing to one o...

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STICHTING NETHERLANDS HEART...

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Project duration: 47 months Date Start: 2023-05-01
End date: 2027-04-30

Project leader

STICHTING NETHERLANDS HEART INSTITUTE No se ha especificado una descripción o un objeto social para esta compañía.

TRL 4-5

Project Budget 7M€

participation deadline Sin fecha límite de participación.

Project description Development of a cure for rare cardiac diseases is a risky, costly and time-consuming process, which is hampered by insufficient insight into pathophysiological mechanisms of the diseases and lack of relevant disease models. The GEREMY consortium proposes to overcome this challenge by developing a gene therapy (GT) for inherited arrhythmogenic cardiomyopathy (ACM), by targeting the disease-causing PLN (non-desmosomes) and PKP2 (cardiac desmosomes) mutations. The GEREMY consortium will apply a unique parallel approach and investigate various promising GT approaches (oligonucleotide chemistries, gene editing and gene delivery). Also, the consortium will engineer disease models for proper assessment of therapeutic interventions and aims to provide in vitro & in vivo preclinical proof-of-concept for the GT.A significant benefit compared to current technologies is that the GT has the potential to be a curative treatment for rare cardiac diseases. Based on previous successes, GEREMY will target PLN & PKP2 as a roadmap of the technology. Restoring the primary defect in the PLN & PKP2 genes that cause the disease will lead to preserving or even restoring myocardial contractility. Through this approach, GEREMY will work towards a curative treatment of inherited ACM and likely other cardiomyopathies, which significantly reduces the healthcare burden. The project’s experts in preclinical research and cardiac genetics will deliver preclinical efficacy and safety data for the mutation correction. The partners with regulatory and clinical trial expertise (EXOM, EUF, NLHI) will prepare for immediate start of clinical trials upon project completion, and apply for orphan drug designation. EUPATI, a patient organisation and KUL ethics partner will ensure that the project is continuously aligned with patient needs and ethical perspectives. The whole consortium will contribute to communicate an disseminate the results to ensure maximum exploitation of this breakthrough technology.

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