Please direct your inquiries and/or proposals

1. Oncology

1-1.Technologies which enhance antibody internalization

  • Not interested in target independent internalization

1-2.Technologies which enable tumor microenvironment-dependent activation of antibodies

  • Not interested in target independent internalization

1-3.Novel/innovative technologies for oligonucleotide delivery and for therapy with non-invasive external force

I.A new delivery vehicle for oligonucleotides into tumors, other than antibody, antibody fragment, peptide and liposome

II.A new technology or an agent which utilizes non-invasive external force (e.g. ultrasonic, radiation and magnetic force, but not limited to those) for cancer treatment and/or diagnosis other than photodynamic therapy 

III.  An innovative and non-invasive photodynamic therapy which is applicable to whole body (any organs, not just surface) for treatment of patients with metastatic tumors

1-4.Novel delivery systems (not liposome nor peptide) for tumor specific expression of contents (DNA/RNA/Protein/Small molecular) 

  • e.g. Bacteria with tumor specificity/orientation
  • e.g. Viruses enabling intravenous injection and multiple dosing

1-5.Protein degradation technologies

I.Novel technologies for identification of “monovalent (non-chimera) protein degrader” or “molecular glue” as an alternative approach for protein degradation

  • Not interested in conventional PROTACs with well-known E3 ligases (CRBN/VHL/IAP/MDM2)

1-6.Targets and technologies for Cell therapy

I.Novel, biologically tractable tumor-associated antigens to which antibodies can be obtained

  • “Antibodies” include monoclonal antibodies, antibody fragments which are applicable to multi-specific antibodies and/or CAR-T
  • “Tumor-associated antigens” include tumor, immune, and stromal cell targets but not limited to those

II.Technologies for Cell therapy

  • Technologies applicable to engineered cell therapies (alpha-beta or gamma delta T cells or NK) which can potentially overcome current issues in treating solid tumor (heterogeneity, immune-suppressed microenvironment)
  • Novel allogeneic technologies which can be applied to the engineered cell therapies

1-7.Novel Immuno-Oncology (IO) therapy and assays

I.Novel assay platform in immuno-oncology research for prediction of clinical efficacy and selection of combination candidates

  • e.g. Ex vivo assay with clinical tumor samples including immune cells

II.Novel IO axis/targets that are not Immune Checkpoint Inhibitors (ICIs). Targets with confirmed mechanisms in human-derived samples are prioritized

  • Research (or targets) for innate lymphoid cells
  • Research (or targets) for molecules specifically expressing on tumor infiltrated myeloid cells
  • Research (or targets) for enhancement of antigenicity selectively in tumor cells
  • Targets/axis which contribute to ICI resistance with clear rationale based on clinical data
  • Small molecules which boost/restore immune systems with strong anti-tumor efficacy in an ICI resistant mouse model in a monotherapy setting

1-8.Novel technologies in IO area

I.Technologies which improve tumor penetration (especially immune cells) either alone or in combination

  • e.g. Tumor microenvironment (TME) specific targeting approach (stroma, soluble factor, etc.)

II.Technologies for multi-specific antibodies to overcome current issues in treating solid tumor (heterogeneity, immune-suppressed microenvironment)

1-9.Drug efficacy evaluation platform reflecting cancer heterogeneity and drug-resistance mechanisms

I.High-throughput, phenotypic-based drug discovery platform reflecting cancer heterogeneity and drug-resistance mechanisms

II.Novel readout technologies other than cell killing applicable for high-throughput screenings are of interest. (ex. Cell painting, high-content image, omics analysis (proteomics, transcriptomics, etc.))

III.  Ex-vivo TME modeling technology reflecting tissue heterogeneity (stromal and immune included) and validated for approved therapeutics.

1-10.Novel approaches for targeting cancer specific nuclear chromatin structure

I.Novel approaches to regulate expression levels of oncogenes/tumor suppressor genes by targeting cancer specific nuclear chromatin structure (ex. DNA-protein, DNA-protein-RNA, super-enhancer)

  • Not interested in nucleic acid-based approach

1-11.Novel approaches (research or targets) for neuron-cancer axis

  • Interested in cancer neuronal crosstalk across all cancer types
  • Priority should be given to certain themes with an evaluation model

2. Rare Diseases

2.1Monogenic Rare Diseases

I.Novel technology of AAV gene therapy for pre-mRNA splicing modulation (e.g. trans-splicing, mRNA repair) or for large genes over the original genome-packaging capacity

II.Novel proteins with beneficial function/character, that were identified by screening or engineering disease-related proteins including other species proteins

  • Not interested in rare cancer

2.2Non-monogenic Rare Diseases

I.Novel therapeutic targets/mechanism for rare refractory end-organ diseases including rare auto-immune diseases based on genetic variants found by GWAS and/or PheWAS analysis or single cell transcriptome analysis (scRNA-seq) from human samples

  • Not interested in rare cancer, non-rare autoimmune disease and symptomatic treatment
  • Targets with clinical validation are preferable

3. Central Nervous System Diseases

3.1Synaptic Dysfunction Disorder

I.Novel targets and evaluation technologies for the treatment of Autism Spectrum Disorder

  • We seek novel molecular targets for the treatment of core symptoms of Autism Spectrum Disorder (ASD), especially by restoring synaptic dysfunction
  • We seek novel in vitro methodologies to evaluate ASD molecular targets or drugs efficacy focus on the changes in the morphology and transmission of the synapses, especially by using human iPS neurons (healthy vs Fragile X Syndrome)

3.2Psychiatric Diseases

I.Technology, device and index for precision medicine for psychiatric disease (patient stratification)

  • Stratification by brain circuit: Research on brain dysfunction caused by abnormalities in specific brain regions and brain circuits
  • Stratification by brain function: Technology, indexes, and analysis methods that accurately and objectively measure human brain function and mental state
  • Vitalization of facial expressions, speech, sleep, etc.: Technologies such as wearable devices that easily and simply substitutes human brain functions

II.New drug targets with highly reliable evidence for Psychiatric diseases

  • Priority diseases include schizophrenia, depression, bipolar disorders, autism spectrum disorders, and genetic diseases, particularly glutamate-targeted research
  • Novel approach to apply existing therapeutic compounds to psychiatric diseases (including changes of administration route, formulation, and endogenous bio-active molecules)
  • Novel targets combined with modality such as peptides and nucleic acids as well as small molecules using DDS technology for brain delivery

3.3Neurodegenerative Diseases

I.Elucidation of mechanisms underlying misfolding of neurodegenerative disease-related proteins such as Amyloid beta, Tau, alpha-syn, PrP, TDP-43, and Htt (polyQ proteins), and their therapeutic approaches

  • Novel approaches for inhibition, restoration and degradation of misfolded proteins as well as generation, identification and isolation of conformational strains
  • Mechanism of intracellular dysfunction influenced by misfolded proteins
  • The clearance mechanism of misfolded proteins from the brain such as glymphatic system

II.Novel technologies for research and diagnosis of neurodegenerative diseases

  • Disease-relevant assay systems and animal models
  • Novel methodologies for diagnosis and stratification of patients based on misfolded protein
  • Objective motor functional measurement applicable to Parkinson’s disease or other neurodegenerative diseases

4. Other Disease Areas

4.1Ophthalmology

I.Novel AAV gene therapy targets and mechanisms for glaucoma and/or for diabetic macular edema (DME)

  • Novel therapeutic targets or mechanisms that have the potential for AAV gene therapy for glaucoma or DME. Focus on early-stage researches such as exploratory research on therapeutic target molecules and technologies. Clinical programs are not interested

4.2Chronic Heart Failure

I.Nucleic acid based therapeutic approaches for chronic heart failure and monogenic cardiac disorder

  • Nucleic acid based therapeutic molecular target and approach (in vitro/ in vivo assay system) for chronic heart failure and monogenic cardiac disorder (dilated/ hypertrophic cardiomyopathy)

4.3Immunology

I.New modality for the induction of antigen-specific immune tolerance

  • New modality which induce the antigen specific Treg cells
  • New modality which induce the specific depletion of antigen presenting cells or the specific transform of antigen presenting cells into immune suppressive cells

5. Technologies

5.1Novel Nucleic Acid Therapeutics

I.Novel delivery platforms or organ selective targeting ligand for antisense oligonucleotides, siRNA or mRNA

II.Novel gene editing technologies unaccompanied by DNA double strand break

III.  Novel molecular targets in genetic disorders suitable for nucleic acid therapeutics (antisense oligonucleotides, siRNA, mRNA, etc.)

5.2Novel Targets for Bispecific Antibody Therapeutics

I.Novel targets and their combinations to show synergistic biological activities or novel biological activities which can be brought by bispecific antibodies, such as activating/inactivating signaling pathways

II.Specific monoclonal antibodies to each target should be available. Fragment antibodies, other scaffold proteins and engineered natural ligand are acceptable as formats of binders

III.  Animal disease models and/or translational research (tissues or cells from patients) are preferable

IV.  Our focus areas are oncology as well as non-oncology such as rare diseases and immune disorders

5.3Novel technology platform to modify or manipulate antibodies/proteins that can be conditionally activated/switched dependent on disease condition or environment

I.Antibodies/proteins which can be activated only with specific disease condition

II.Specific diseases include tumor, rare diseases and immune disorders

III.  The biological conditions include concentration of certain molecules, pH (for non-oncology only), and temperature etc.

IV.  Proteins include cytokine or protease

5.4Novel technologies for engineering rAAV vectors

I.Technologies enabling multiple administration of rAAV vector to the same patient

II.Technologies enabling strict regulation of gene expression for rAAV vectors

III.  Technologies utilizing existing methods such as tet-on/off are out-of-scope

IV.  Technologies enabling organ/cell-type specific infection of rAAV (including utilization of the novel capsid), especially for CNS area

5.5Technologies for identifying hit/lead peptides that possess both cell-permeability and target binding activity for regulation of intracellular protein-protein interaction

I.Technologies enabling multiple administration of rAAV vector to the same patient

II.Methods to quantitatively analyze the entry of the peptides into cells are also needed

III.  In silico technologies to predict and design permeable peptide like molecules

  • Not interested in fusions with known cell-penetrating moiety such as CPP (e.g. R8, TAT)

5.6Novel technologies for RNA/DNA-targeted drug discovery

I.Technologies enabling screening of small molecules or peptides that modulate RNA splicing, non-coding RNA processing or expression of gene of interest

II.Biophysical, biochemical or in silico technologies related to analysis or prediction of small molecule-RNA/DNA interaction or RNA-protein interaction

5.7Proteases or ion-channels as therapeutic targets

I.Extracellular proteases or ion-channels to be shown or expected as a main factor for diseases.

  • Not interested in target fishing research that the relations between target molecules and diseases are still unclear

5.8Drug Delivery System (DDS) Technologies

I.Novel cancer-specific delivery platforms for chemical compounds, antisense oligonucleotides and biologics

  • Not interested in molecular modification and conjugation with antibody or synthetic polymer

II.Novel DDS technologies for tissue specific targeting

  • Target tissues: CNS, heart, muscle, lung or kidney
  • Novel carriers or DDS technologies to deliver drugs to the tissues using physiological mechanisms
  • Not interested in technologies which physically open the BBB

III.  Novel DDS technologies for enhancing drug permeability

  • Examples of the technologies: enhancement of oral absorption of peptides and macromolecules, improvement of percutaneous absorption of low and middle molecule compounds
  • Not interested in technologies which physically open the tight junction

5.9Bioanalysis

I.Novel bioanalysis technologies for new modality ADME research

  • Novel bioanalysis technologies to characterize and quantitate biotransformation of biologics (antibodies, ADC, etc.)
  • Novel technologies to engineer bioanalytical reagents that selectively capture or detect new modalities (diverse biologics, oligonucleotides, peptides, etc.)
  • Not interested in well known methods (conventional LBA, LC/MS, conventional preparation of monoclonal/polyclonal Ab, etc.), although it is acceptable if remarkable progress is expected

5.10In silico technologies to support small molecule drug discovery

I.Novel in silico drug discovery technology using ONLY sequence information

  • Novel methods for virtual screening, ligand estimation and drug design using ONLY sequence information
  • e.g. protein structure prediction, binding site estimation, or machine learning-based activity prediction

II.Novel molecular representation technology to predict parameters (bioactivity / ADMET) of compounds using machine learning and deep learning

  • Proposed technology should outperform commonly used technologies such as ECFP4/6 or molecular graph (i.e. DeepChem)
  • For example, chemical descriptor, graph, quantum chemistry, molecular dynamics or any other method
  • The technologies requiring high computational cost are out of scope

5.11Hit finding and hit to lead technology for small molecules

I.Technology of creating unique chemical library

  • Methods for creating chemical library using untraditional chemical reactions, such as electrochemical reactions, photochemical reactions, enzymatic reactions etc.

II.Novel hit / lead finding technology targeting “undruggable” proteins or RNAs

  • Method or technology of identifying hidden binding pockets (cryptic binding pockets) for small molecules on “undruggable” proteins or RNAs
  • Covalent drug discovery technology
  • Binder screening technology

III.  Technology of small molecule drug delivery to cancer cells

  • Except for technology of enzyme prodrug therapy, pH-dependent drug delivery

IV.  Novel technology of CNS drug delivery

  • Small molecules drug delivery technology, such as prodrug technology and small molecule-drug conjugate to deliver the small molecules to central nervous system

5.12Safety

I.Prediction of immunogenicity of biopharmaceuticals for humans

  • In vitro methods different from T-cell proliferation assay using CD8 positive T-cell depleted hPBMC, or in vivo methods using a humanized animal model and so on

II.Non-clinical evaluation models to predict clinical immune-related AE

  • To detect clinical immune-related adverse effects by drugs that modulate immune checkpoints or mediate immune reactions