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

Research on cellular senescence in neurodegenerative and neurodevelopmental diseases

Calling for research that can contribute to the understanding of involvement of cellular senescence and to future drug discovery targeting cellular senescence in central nervous system diseases.

Areas of interest:

• Clarification of the relationship between cellular senescence (including genomic instability, leakage of dsDNA and mtDNA, and cell phenotype) and neurodegenerative and neurodevelopmental diseases
• Neurodegenerative and neurodevelopmental diseases such as rare diseases, Parkinson's disease, Alzheimer's disease, and autism
• Priority is given to research using human samples, particularly patient samples, for future clinical applications
  
  

#3

Research on pathophysiology of blood-brain barrier in neurodegenerative diseases

Areas of interest:

• Neurodegenerative diseases such as rare diseases, Parkinson's disease, and Alzheimer's disease
• Study aiming for therapeutic applications through functional analysis of blood-brain barrier in neurodegenerative diseases
• Priority is given to proposals that analyze clinical data and samples
  
  

#4

Research on non-demented with Alzheimer's neuropathology

Areas of interest:

• Elucidation of molecular mechanisms to confer resistance to cognitive decline
• Study to identify therapeutic targets or stratify patients based on molecular mechanisms of non-demented with Alzheimer's neuropathology
  
  

#5

Research on cell therapy for central nervous system

Areas of interest:

• Neurodegenerative diseases such as rare diseases, Parkinson's disease, and Alzheimer's disease
• Study for microglia cell therapy through peripheral delivery for the above-mentioned diseases
• Microglia delivery method establishment to central nervous system
  
  

#6

Research on physiological and pathological significance of iron accumulation in neurodegenerative diseases

Areas of interest:

• Neurodegenerative diseases such as rare diseases, Parkinson's disease, and Alzheimer's disease
• Study regarding the impact of iron accumulation on neuronal or synaptic function for the above-mentioned diseases
• Study regarding iron and ferroptosis
• Novel therapeutic approach to prevent neurodegenerative diseases associated with impaired ferritinophagy
  　・New neurodegenerative diseases involving ferritinophagy
  　・New methods for detecting ferritinophagy at the cellular level
  　・In vitro/in vivo ferritinophagy-related biomarkers and compounds that regulate ferritinophagy
  
  

#7

Ideas on novel small molecule therapeutics for congenital muscle diseases such as muscular dystrophies (DMD, DM1, etc.) or congenital myopathies

We are taking applications for ideas to identify novel small molecule compounds that can directly intervene in the causal gene or the pathogenic mechanism based on the genetic mutation for the treatment of congenital muscle diseases. Symptomatic relief approach is out of our focus.
It is preferable if the proposal includes platforms or ideas to predict the clinical effects of the compound appropriately. For example, functional evaluation systems using patient-derived cells such as iPSCs or animal models with precise genetic-features of the disease, etc.

#8

Ideas on novel small molecule therapeutics for hereditary neuropathies including Charcot-Marie-Tooth (CMT) disease, spinal and bulbar muscular atrophy (SBMA), or congenital myasthenic syndrome (CMS), etc.

We are taking applications for ideas to identify novel small molecule compounds that can directly intervene in the causal gene or the pathogenic mechanism based on the genetic mutation for the treatment of hereditary neuropathies. Symptomatic relief approach is out of our focus.
It is preferable if the proposal includes platforms or ideas to predict the clinical effects of the compound appropriately. For example, functional evaluation systems using patient-derived cells such as iPSCs or animal models with precise genetic-features of the disease, etc.

#9

Establishment of neuroinflammation related cellular model/animal model and strategy for patient stratification for inhibitor of innate immune system

Innate immune systems has been suggested as the key pathogenic players in neurodegenerative disorders. Among such innate immune systems, we are interested in cytosolic DNA-sensing, inflammasome and complement activation. We prioritize following proposals:

• A research of cytosolic DNA-sensing, inflammasome or complement activation.

In particular:

• Neuroinflammation related cellular model/animal model
• Strategies for patient stratification for inhibitor of innate immune system.

In case Takeda possesses innate immune system inhibitors which an applicant want to utilize, he/she can utilize them.

#10

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

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
• 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
  
  

#11

Novel methods to enhance clearance system of abnormal proteins from brain, glymphatic system

In many of neurodegenerative disorders including Alzheimer's disease, accumulation of abnormal proteins in the brain is the main pathological change. As a cause of such accumulation, dysfunction of glymphatic system, a brain drainage system, that removes such abnormal proteins from the brain, has attracted attention in recent. Therefore, restoring the dysfunction of this glymphatic system is extremely important to prevent or delay the progress of various neurodegenerative disorders. We want novel innovative basic researches to address that issue.

Areas of interest:

• Neurodegenerative diseases such as rare diseases, Parkinson's disease, and Alzheimer's disease
• Elucidation of cellular waster clearance mechanisms in the brain
• Priority is given to proposals that analyze clinical data and samples
  
  

#12

Drug discovery research and Translational research for white matter damage in neurodegenerative disease

Here we call for the idea that materials obtained from patients whose diseases underlie white matter damage are applied for the drug discovery research and the translational research. Proposes for the Omics analyses of white matter damage lesion and/or for the disease modeling that recapitulates neuron-glia interaction with patient-derived cells are welcomed. If needed, technical supports which are necessary to conduct proposed studies may be provided from our research organization upon requests. Our focus areas are Alzheimer’s diseases, Frontotemporal dementia, and rare diseases, such as leukoencephalopathy.

Other Areas of Interest

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