Neuroscience

(Please refer “Platform technology” section for neuroscience related subjects)

#1

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.

Prioritized proposals:

• A proposal of novel drug target and its validation study plan which is originally established by NGS-data analysis of clinical samples or public human data sets.
• Feasibility validation plan* with high predictability of clinical efficacies. *Utilization of translatable preclinical models (e.g. patient iPSCs), and translatable readouts related with disease prevention/therapy.

Area of interest:

• Amyotrophic lateral sclerosis (ALS), Huntington's disease (HD) and related triplet diseases
  
  

#2

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:

• Elucidation of molecular mechanism of disease, omics analysis, or establishment of drug efficacy evaluation system using patient-derived clinical samples
• Research with clinical symptoms and molecular pathology that leads to drug targets or patient stratification
  
  

#3

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.

Prioritized proposals:

• A proposal of novel drug target with high clinical evidence which is originally established through analysis of clinical samples or public human data sets, such as NGS
• A proposal of novel biomarkers which is clinically detectable (e.g. blood components, MRI imaging)

Notes:

• Do not include ALS, Huntington’s disease, frontotemporal dementia, Down syndrome with cognitive decline, postoperative cognitive dysfunction, psychiatric disorder, stroke, pain, migraine and multiple sclerosis (relapsing-remitting type)
• No constraint on modality (small molecule, nucleic acid, antibody, etc)
  
  

#4

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

Prioritized proposals:

• A proposal of definition of disease and patient classification
• A research of biomarkers which can stratify patient population

Examples of approach to prevent or recover age-related disturbance of neuronal function:

• modulation of sleep quality and circadian rhythm: including research about short sleeper
• maintenance of sensory system: including hearing loss
• waste clearance mediated by glymphatic system or autophagy
• maintenance and recovery of blood-brain barrier integrity
• regulation of age-related inflammation
• functional recovery of mitochondria or maintenance of organelle homeostasis
• inhibition or decrease of accumulation of DNA damage

Areas of no interest:

• exercise therapy
• nutrition therapy
  
  

#5

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.

#6

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:

• Establishment of animal and cellular model that mimic disease states and symptoms
• Construction of drug efficacy evaluation systems using clinical specimens such as patient-derived blood, fibroblasts, urine, and various tissue stem cells (including technologies for direct differentiation and rapid differentiation into target cells.)
• Target and technology for next generation gene therapy
• Technology on drug brain penetration
  
  

#7

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.

Other Areas of Interest

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