MusclePlate
Financiado
Human skeletal muscle platform for disease modelling and high-throughput drug sc...
Neuromuscular disorders (NMDs) are a collection of pathologies that affect the skeletal muscle (SkM) function and lead to reduced mobility, breathing issues and death. Most NMDs are incurable and existing therapies are generally i...
Neuromuscular disorders (NMDs) are a collection of pathologies that affect the skeletal muscle (SkM) function and lead to reduced mobility, breathing issues and death. Most NMDs are incurable and existing therapies are generally ineffective. The major contributor for the lack of NMD therapies is the absence of human NMD research models, applicable for the screening and validation of therapeutic candidates. Our goal for this project is to develop an in vitro platform containing highly biomimetic and functional SkM analogues, where NMDs can be modelled with the aim of testing drug candidates, in a reliable, high-throughput (HT), and quick manner. The platform will consist of 96 individual 3D tissue units, generated from the myogenic differentiation of human induced pluripotent stem cells (hiPSCs). To induce biomimetic and mature tissue formation, the platform will combine a unique topographical design with a light-stimulation system for optogenetic control of SkM contraction. Using image-based assays and computational algorithms, we will be able to automatically extract and quantify the SkM phenotypical (e.g. intracellular structure) and functional (e.g. contraction speed/force) parameters. To show NMD modelling ability within the platform, we will knockout BIN1, a gene whose mutated form is involved in the onset of centronuclear myopathy (CNM), a fatal and incurable NMD. The validity of the NMD model as a drug discovery platform will be demonstrated by testing compounds with known therapeutic activity. This proof of concept project will lay the ground work for a spin-off company devoted to HT drug screening for NMDs.
The successful development of this platform will provide an invaluable tool for pharmaceutical research, allowing accurate, convenient and cost-effective identification of novel drugs and therapeutic targets for NMDs and, ultimately contributing to the acceleration of novel therapies for NMD patients.
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Duración del proyecto: 20 meses
Fecha Inicio: 2023-02-22
Fecha Fin: 2024-10-31
Fecha Fin: 2024-10-31
Línea de financiación: concedida
El organismo HORIZON EUROPE notifico la concesión del proyecto el día 2024-10-31
Línea de financiación objetivo
El proyecto se financió a través de la siguiente ayuda:
ERC-2022-POC2:
ERC PROOF OF CONCEPT GRANTS2
Cerrada
hace 2 años
Presupuesto
El presupuesto total del proyecto asciende a
150K€
Líder del proyecto
INSTITUTO DE MEDICINA MOLECULAR JOAO LOBO ANT...
No se ha especificado una descripción o un objeto social para esta compañía.
Perfil tecnológico
TRL
4-5
Perfil tecnológico
TRL
4-5