Neuroscience and relevant platform technologies

#1

Innovative therapeutic approaches to the below-listed diseases in neuroscience area, including but not limited to

• Novel drug targets or patient stratification/treatment-prediction biomarkers
• Generation of hit or lead compounds with pharmacological data based on an innovative drug discovery approach and/or mechanism (with a preference for those supported by in vivo data)
• In vitro/in vivo small molecule imaging probes for disease diagnosis and patient stratification

• Amyotrophic lateral sclerosis
• Frontotemporal lobar degeneration
• Alzheimer’s disease
• Parkinson’s disease
• Multiple system atrophy
• Cerebral amyloid angiopathy
• Huntington’s disease
• Ataxias
• Myotonic dystrophy type 1
• Duchenne muscular dystrophy
• Facioscapulohumeral muscular dystrophy
• Charcot-Marie-Tooth disease type 1A
• Myasthenia gravis
• Sarcopenia
• Epilepsy
• Repeat expansion diseases other than those listed above

#2

Proposals addressing novel disease mechanisms that will enable highly innovative therapeutic approaches or reclassification of CNS diseases, based on human pathophysiology or physiology

Approaches of high novelty such as those addressing

• Neuropathy due to autoantibodies or autoimmunity
• Waste clearance from the brain
• Blood-brain barrier integrity
• Organelle homeostasis
• DNA damage
• RNA dysregulation
• Resistance/protective factors against diseases (i.e. resilience)
• Liquid‒liquid phase separation
• Aberrancy in glial cells

The following diseases are out of focus:
Multiple sclerosis, Acute ischemic stroke, Acute phase of traumatic brain injury, Cerebral thrombosis, Cerebral hemorrhage, Spinal cord injury, Pain, Neuromyelitis Optica

#3

Novel platform technologies to accelerate nucleic acids and small molecule drug discovery within neuroscience area

1. Development and validation of novel platform technologies on nucleic acid medicines
• Novel approaches to improve efficacy, selectivity, toxicity profile and/or durability of nucleic acid medicine (e.g., new nucleotide modifications, new nucleotide constructs, etc.)
• DDS (drug delivery system) technologies for tissue-selective transportation of nucleic acid medicines (e.g., technologies to improve blood-brain barrier/blood-nerve barrier penetration, intrathecal delivery technology to the deep brain, de-targeting specific organ distribution, and devices that enhance drug delivery)
• Technologies for predicting in vivo PK/PD of nucleic acid medicines
2. Development and validation of novel platform technologies on synthetic chemistry
• Innovative synthetic chemistry technologies (e.g., practical chemical reactions with high selectivity and yield (e.g., photo-redox reaction, electrochemical reaction, flow reaction, etc.), bio-orthogonal chemical reactions, and new conjugation technologies, etc.)
• Versatile technologies to improve physicochemical properties, such as solubility, of therapeutic compounds (Co-crystal, host-guest complex, etc.)
3. Applications of AI (artificial intelligence), ML (machine learning), molecular simulation, and automation technology in drug discovery
• Optimization of chemical reactions, synthetic route and scale-up synthesis
• Methodologies for predicting pharmacological activity and potential side effects of the compounds, and methodologies for identification of novel MoA (mode of action)
• Technologies for virtual/in silico target screening
• Discovery of novel biomarkers with high translational confidence

Other Areas of Interest

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