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Platform technology (assay systems and analysis methods)

#16

Development of a phenotypic assay to reflect neurodegenerative disease phenotypes


Those phenotypic assays including the following elements will be prioritized.

  • Neurodegenerative disease-oriented such as rare neurodegenerative diseases, Parkinson’s disease, or Alzheimer’s disease
  • Direct conversion of patient fibroblasts or other somatic cells into neurons
  • Target identification and functional analysis of the direct conversion
  • iPSC-derived neuronal assay systems adding aging factors
  • Cell panel development to cover heterogeneous disease phenotypes



#17

Novel technology platform to realize precision medicine by using patient-derived somatic cells for neurodegenerative diseases


iPSC-based cell models are often used for studying disease mechanism and discovering drug candidates for human diseases including neurodegenerative diseases. However, drawbacks of iPSC-based cell modeling include high cost, time consuming workflow, and labor intensiveness. In this research project, we will look for novel technology platform that ultimately enables precision medicine by using patient-derived somatic cells. A platform includes differentiation methods to generate cell types of interest involved in neurodegenerative diseases from somatic cells, cell-based assay in differentiated cells, in vitro studies for biomarkers and drug discovery.



#18

Novel therapeutic approach or assay development for neurodegenerative diseases caused by mitochondrial dysfunction or damaged mitochondria


Mitochondrial dysfunction and abnormal clearance (mitophagy) are one of the factors that cause and exacerbate neurodegenerative diseases. In addition, inflammatory responses caused by leakage of mtDNA and other factors from damaged mitochondria have recently attracted attention. Here, we call for the ideas about new drug targets or therapeutic concepts for neurodegenerative diseases especially focusing on damaged mitochondria or mitochondrial dysfunction, as well as research tools (e.g., disease modeling and sophisticated evaluation systems for mitochondrial dynamics and function) that can be used for these studies.



#19

Research for the common mechanism of the protein aggregations in proteinopathies, and assay development for an evaluation system for exploring aggregation inhibitors


Neurodegenerative diseases, such as ALS and PD, are classified into a group of diseases called proteinopathies, which have a common mechanism of neuronal cell death caused by the accumulation of toxic aggregated proteins such as TDP-43 and α-synuclein in neurons by forming abnormal protein structures. Recently, the novel concept of aggregation mechanism by which these proteins acquire pathogenicity is through condensed structures contained in droplets separated by liquid-liquid phase separation (LLPS), followed by the transition to highly pathogenic fibrotic structures was proposed. Based on this hypothesis, we look for the research to elucidate the molecular mechanism by which aggregating proteins undergo morphological transition to become toxic conformation, and the evaluation systems for protein aggregation formation and dissociation that apply this mechanism.



#20

Biomarker research of patient stratification for neuro-degenerative diseases; e.g. ALS, rare disease, FTD, AD


Neurodegeneration has been observed in specific cell types or regions of brain in patients, such as ALS, FTD, and AD, which have diversity with causal genetic mutation and pathologies. However, biomarkers for patient subtype and stratification have not been fully established. We would like to recruit proposals in terms of novel biomarker research for patient stratification and their drug response for neurogenerative diseases, including assay development using biofluid samples and cutting edge science reflecting disease specific phenotype (eg. RNA splicing, post-translational modification, and liquid-liquid phase separation).

Areas of interest:

  • Amyotrophic lateral sclerosis (ALS), rare diseases, frontotemporal lobar degeneration (FTLD), Alzheimer's disease (AD)



#21

Biomarker and drug target research for novel non-coding RNA reflecting specific disease mechanism or disease progression for neuro-degenerative disease


Non-coding RNA (ncRNA) such as lncRNA, miRNA, and cirRNA has been recently highlighted as a biomarker (BM). Multiple ncRNAs involving in pathological conditions have been reported, however, little is known about its biological mechanism and practical BM possibility. Here, we call for the idea about RNA biomarkers / possible drug targets reflecting the disease mechanism or specific pathological conditions. Especially, analysis for novel RNA biomarkers in plasma or cerebrospinal fluid, development of quantitative assay, and mechanism of action analysis for the identified RNA biomarkers are applicable.

Areas of interest:

  • Amyotrophic lateral sclerosis (ALS), rare diseases, frontotemporal lobar degeneration (FTLD), Alzheimer's disease (AD)



#22

Development of next-generation organoids using engineering approaches


The rise of human organoid technology has developed mini-organs that have complex cell-cell interactions with the immune system, nervous system, vascular system, etc. and can mimic the development and pathology. Also, It is becoming possible to construct a disease model involving multiple organs such as Parkinson's disease (brain-intestine) and non-alcoholic hepatitis (brain-liver). However, in order to truly imitate the development and pathophysiology of these next-generation organoids and apply them to drug discovery, it is necessary to give mechanical stimuli such as perfusion, oxygen pressure, and extracellular matrix on the culture dish, and to improve the organoid culture by integrating big-data from multi-omics, single-cell analysis, and imaging. Therefore, in this project, we aim to explore cutting-edge technologies and ideas including physical and information engineering approaches for the development of next-generation organoids.

Keywords:

  • Organoid × biomechanics: 3D printer, organ-on-chip, perfusion
  • Organoid × Informatics: AI, omics, single-cell analysis, imaging



#23

Research on preclinical models or clinical biomarker discovery for inherited muscle diseases and cardiomyopathies


We are seeking ideas for preclinical model studies and clinical biomarker discovery studies that are essential for drug discovery research for inherited muscle diseases and cardiomyopathies for which there are no effective treatments.

Examples of research:

  • Research on non-clinical models of inherited muscle diseases and cardiomyopathies with high extrapolation to clinical practice (e.g., pathological analysis research using animal models and in silico pathological model research).
  • Research to identify novel clinical biomarkers (including imaging) that can predict disease progression or drug efficacy in a minimally invasive and non-invasive manner in patients with inherited muscle diseases and cardiomyopathies.



#24

Production technology and quality verification of human ES/iPS cell-derived brain organoids


Since brain organoids have a potential to reflect brain functions closer than two-dimensional neuronal cultures, they would be expected to be applied to a wide range of researches such as disease biology and drug discovery for central nervous system diseases. High quality productive technology of the brain organoids is indispensable to achieve our expectation. Here, we call for research ideas and technologies on the ES/iPS cell-derived brain organoid production and its quality verification. It is desirable to be a proposal with a future perspective involving the establishment of brain organoid disease model and/or organoid-based drug discovery using neural function as an index.

Target diseases:

  • Autism spectrum disorder, epilepsy, sleep disorder, depression etc.



Other Areas of Interest


How to Apply

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CONTACT

Please contact the COCKPI-T® Funding Office for further information.

COCKPI-T@takeda.com

  

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