(Please refer “Platform technology” section for neuroscience related subjects)
Proposal and validation study of novel therapeutic approaches for rare/intractable diseases associated with neurodegeneration; e.g. ALS, Huntington’s disease and ataxias
New target proposal of innovative modalities (e.g. small molecule, nucleic acid, antibody) for signature features including abnormal RNA/DNA structures, RNA splicing and protein production observed widely in rare/intractable neurological diseases.
Area of interest:
Molecular mechanism research of disease pathology or reverse translational research with clinical data and samples in frontotemporal dementia, Down syndrome with cognitive decline, or postoperative cognitive dysfunction
Calling for molecular mechanism study and reverse translation study in the described diseases. Priority is given to the following research that is linked to clinical symptom and pathology as well as to drug discovery using self-acquired or published clinical data and samples.
Areas of interest:
Proposal of novel therapeutic approaches and validation study for neural disorders which do not fall upon the above (#1 and #2)
Novel therapeutic approaches and validation study proposal for neural rare disease and neuromuscular disorders other than indications depicted in #1 and #2.
Novel therapeutic approach to prevent or recover age-related disturbance of neuronal function; e.g. sleep/circadian rhythm, waste clearance, BBB integrity, inflammation, organelle homeostasis, DNA damage, sensory system
Examples of approach to prevent or recover age-related disturbance of neuronal function:
Areas of no interest:
Definition of novel disease concepts based on biomarker strategy for strict patient stratification, followed by establishment of pathophysiologically-relevant cellular model, and therapeutic approach including gene therapy
Neurodegenerative and neurological disease have been diagnosed by their representative phenotypes. But among them, pathologies and etiologies are so diversified from the scientific point of view, and that makes it very difficult to discover therapeutic drugs. Due to the heterogeneity of these diseases, explored compounds by focusing on one aspect and phenotype has little success rate against whole overall patient population, strict redefinition of disease concepts that share common biological and medical characteristics within small restricted patient population is essential for successful clinical trials. In other words, stratification strategies such as genetic testing and biomarker quantification for defining novel specific homogeneous disease seems to be critical. In this program, we are recruiting for strategies to achieve uniform patient population stratification, and cell model establishment and therapeutic drug discovery for stratified patient population.
Development of new research tool, drug assay system or drug modality for neurodegenerative disease or rare disease with neurodegenerative pathology
Calling for novel and original research tool, assay systems to evaluate drug efficacy, and drug modality for diseases involving neurodegeneration
Areas of interest:
Novel drug targets, therapeutic concepts and research tool for aberrant glia-neuron network involved in neurodegenerative diseases such as FTLD, PD, ALS, AD.
Malfunction of glial cells plays detrimental role in the progression of neurodegenerative disease, and better understanding of aberrant glia-neuron network to improve therapeutic intervention is desired. Glia-neuron interaction are known to involve secretory factors and synaptic modulation, but recently, for example, somatic junction that signals from neuronal mitochondria to microglia has been reported, implicating that there is a broad mechanisms and pathways. Here we call for the idea about new drug targets, therapeutic concepts and/or research tools that drive investigation on the mechanisms which involve aberrant glia-neuron network in association with cell-autonomous malfunction in neurodegenerative diseases such as FTLD, ALS, PD, and AD.
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