Hola,
¿eres nuevo aquí?

Regístrate gratis y conecta tu empresa con financiación pública, partners y proyectos.

Tengo cuenta

Regístrate

Ver video

GEREMY

Financiado

Cerrado

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... ver más

30/04/2027

STICHTING NETHERLA...

7M€

Presupuesto del proyecto: 7M€

Líder del proyecto

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

TRL 4-5

Fecha límite participación Sin fecha límite de participación.

Financiación concedida El organismo HORIZON EUROPE notifico la concesión del proyecto el día 2023-05-01

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

I+D

Cerrada hace 3 años

0% 100%

Información adicional privada

No hay información privada compartida para este proyecto. Habla con el coordinador.

Participantes

STICHTING NETHERLANDS HEART...

Lider

Conecta tu I+D

¿Tienes un proyecto y buscas un partner? Gracias a nuestro motor inteligente podemos recomendarte los mejores socios y ponerte en contacto con ellos. Te lo explicamos en este video

Proyectos interesantes

Edit-hCOs Precise Genome Editing to Correct Cardiomyopathies in Human... 189K€ Cerrado

HCMSpliceAOC Molecular design and application of splice-switching antibod... 157K€ Cerrado

Cor-Edit-P Cardiac open reading frame edition to study cardiomyopathies... 2M€ Cerrado

PID2020-116935RB-I00 ESTUDIO DE LA FUNCION DE PKP2 EN ENFERMEDADES CARDIACAS, ANA... 198K€ Cerrado

TRACTION Transcription Factor Gene Therapy for Bradyarrhythmias 2M€ Cerrado

CardioReGenix CardioReGenix Development of Next Generation Gene Therapies... 15M€ Cerrado

Duración del proyecto: 47 meses Fecha Inicio: 2023-05-01
Fecha Fin: 2027-04-30

Líder del proyecto

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

TRL 4-5

Presupuesto del proyecto 7M€

Fecha límite de participación Sin fecha límite de participación.

Descripción del proyecto 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.

Conecta tu I+D

Entra hoy

¿Olvidé mi contraseña?

Financiación

Empresas

CTIs/Universidades

Proyectos

Investigadores