Platform technology (new modalities and drug delivery systems)

#19

Chemistry-driven novel methods or ideas to bring breakthrough into CNS drug discovery

We recruit a small molecule-based approach to a CNS therapeutic target. The followings are examples.

• Drug discovery research on tough targets
  (e.g.) Biased- or heterodimer-selective GPCR ligands, Allosteric ligands, Phosphatase inhibitors, Protein-protein interaction modulators

• Drug discovery research based on innovative mechanism of action
  (e.g.) Protein degradation inducer, Ligands acting on DNA/RNAs (incl. splicing modulators), Small molecule chemical chaperones

• Drug discovery research based on polypharmacology
  (e.g.) Research on combination of therapeutic targets in terms of chemistry and pharmacology, Effective methodology for compound optimization.

#20

Chemistry-driven novel technologies, methods or ideas to solve issues on drug discovery with new modalities

We recruit research themes to solve issues in drug discovery using new modalities. The followings are examples.

• Investigation of technologies for the improvement of efficacy/biodistribution of antisense oligonucleotides and siRNA
• Research on Structure-Activity/Toxicity-Relationship in oligonucleotide drug discovery based on 3D structural analysis
• Research on brain-targeted drug delivery technologies and evaluation of their mechanisms of action
• Research on muscle tissue- and/or cell-targeting drug delivery technologies and evaluation system of their mechanism of action
• Drug discovery research based on regenerative medicine/phenotypic screening
  (e.g.) Novel methods for target identification. Research on small molecules to facilitate cellular process.
  
  

#21

Chemistry-driven novel technologies, methods or ideas to improve and/or accelerate drug discovery process

We recruit research themes to improve and/or accelerate the drug discovery process. The followings are examples.

• Development of novel drug discovery algorithms based on artificial intelligence and/or molecular simulation
  (e.g.) Pharmacological activity and/or adverse effect prediction. Design of novel compounds

• Development of a cheminfomatics platform to manage a huge amount of information in combination with artificial intelligence and/or molecular simulation
  (e.g.) structure search for a complex molecule such as an　oligonucleotide

• Development of technologies to create next generation diverse compound libraries
  (e.g.) Novel libraries designed for CNS therapeutics, phenotypic screening

• Development of in vitro / in vivo imaging probes for disease diagnosis, patient stratification
  
  

#22

Capsids with defined tissue tropisms (e.g. skeletal muscle) or capsids with reduced immunogenicity

Current viral capsids that are utilized in gene therapies (e.g. adeno associated virus [AAV]) tend to have a non-specific tissue tropism and induce a significant immune response post administration. In order to refine the delivery of next generation gene therapies the identification of capsids that preferentially target specific tissues and/or do not induce an immune response would potentially allow lower doses of vectors and/or allow dosing of patients with pre-existing immunity or allow re-dosing of previously treated patients.

#23

Development of novel delivery technology for gene therapies

Recently, several AAVs are used as intravenous injection-enabled DDS for gene therapies, however, those have the issue of administration frequency due to the immunogenicity of AAV itself.
Here, we are seeking proposals for development of novel delivery materials or devices for gene therapies containing mRNA, siRNA or sgRNA meeting following conditions,

1) Non-viral based

2) Systemically available

3) Repeatedly available

4) Possess tissue tropism, particular skeletal muscles and heart

Furthermore, we are seeking novel technologies that can enhance delivery efficiency of existing DDS materials (e.g., novel CPP, hydrodynamic injection or bubble liposome and so on).

Other Areas of Interest

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