Platform Technology-oriented Proposals

Oncology

REF#6

Translational research platforms for immuno-oncology

The following topics will be prioritized:

• Translationally-relevant humanized animal models and/or in vitro/ex-vivo tumor organoid systems reflecting human immune components
• Novel biomarker discovery approaches/platforms for immuno-oncology

Rare Diseases (Genetic and Hematology)

REF#7

Imaging agents of lysosomal health

Approaches enabling visualization of lysosomal activity in vivo are envisioned. Small molecules that can be labeled for PET imaging are of particular interest.

Medicinal chemistry

REF#8

Novel technologies for nucleic acid drug development

Chemistry-driven technologies focusing on the following topics will be prioritized

• Novel approaches to improve efficacy, selectivity, toxicity and/or durability of nucleic acid medicine (e.g. new nucleotide modifications, new nucleotide constructs)
• Technologies that confer nucleic acid medicines with new mechanism of action (e.g. RNA editing, gene up-regulation)
• Delivery technologies (e.g. novel approaches to enhance tissue-specific delivery, detargeting of kidney and liver, novel delivery vehicles)

REF#9

Chemistry-driven novel technologies, methods or approaches to accelerate drug discovery process

The following topics will be prioritized:

• Novel platform technologies for protein degraders or DNA/RNA targeting ligand
• Application of AI/machine learning/molecular simulation to drug discovery (e.g. methodologies for identification of hit molecules, novel MOA, and/or target screening)
• DDS (drug delivery system) technologies for tissue-selective transport of middle/high-molecular weight molecules (including technologies to improve oral absorption or detargeting specific organ distribution)
• Innovative synthetic chemistry technologies (e.g. synthesis of challenging building blocks, novel conjugation technologies)
  
  

DMPK

REF#10

Development of biosensors for pharmacokinetic evaluation

Platforms that enable the following will be prioritized:

• Drug monitoring in microenvironments such as capillaries and CSF space
• Evaluation of the existence form of drugs

Safety

REF#11

Establishment of human glomerular microphysiological system (MPS) that can predict glomerular damage and glomerular filtration function

• in vitro microphysiological system (MPS) that can faithfully recapitulate physiological human renal function to predict safety and pharmacokinetics of drugs in the kidney
  In particular, development of a human glomerular MPS that can assess glomerular tissue damage and filtration function.

REF#12

Development of novel devices and analytical approaches to evaluate pathophysiological conditions or drug toxicities in animal studies

Proposals addressing innovative approaches for measuring biological parameters to evaluate pathophysiological conditions and drug toxicity in animal studies (rats, mice, dogs, monkeys, etc.) will be considered. Specifically,

• Development of 1) devices, 2) software, or 3) experimental methods to acquire and analyze biological parameters such as blood pressure, biochemical parameters, emotional and behavioral characteristics
• Investigation of methodologies already employed in humans with laboratory animals, with the aim of analyzing underling mechanisms and versatility, may also be of interest

REF#13

Development of novel methodologies for evaluation and/or prediction of RNA binding specificity of small molecules

Proposals that will contribute to reducing safety concerns of RNA-targeting small molecules will be considered. Specifically,

• Experimental methods and in silico prediction platforms to evaluate RNA binding specificity of small molecules
• Platforms that allow in silico analysis of RNA structures amenable to ligand binding may also be of interest