Crispr Therapy for Dentatorubral-Pallidoluysian Atrophy (DRPLA)

Upstream Stop Codon Insertion to Suppress CAG Repeat–Driven Disease Progression

C11orf97: A Novel Cancer-Specific Target Inducing Apoptosis

Development of Molecular Targeted and Nucleic Acid Therapeutics for C11orf97

Cancer Metastasis Inhibitor

Target molecules and their inhibitors that control the survival and reactivation of disseminated tumor cells (cancer metastasis-initiating cells).

Enhanced mRNA Expression Platform Using Synthetic 5’UTR Repeat Sequence

Inserting 50-nucleotide synthetic repeat sequences into the 5’UTR significantly enhances mRNA translation, achieving up to 10-fold increases in protein expression in both cells and animals

Macrophages as Drug Targets for Hypertension Treatment

Discovery of a new mechanism of hypertension induction and proposal for drug discovery collaboration based on the mechanism.

Treatment of heterotopic ossification in patients with fibrodysplasia ossificans progressiva (FOP)

New therapeutic targets and therapeutic agents for trauma-induced heterotopic ossification in FOP patients.

Type A CpG Oligo-Containing Lipid Nanoparticles for Cancer Immunotherapy

D35LNPs stimulate TLR9 and significantly induce the release of IFN-α from plasmacytoid dendritic cells, thereby strongly enhancing systemic immunity

siRNA medicines for polyglutamine disease

siRNAs containing innovative artificial nucleic acids selectively suppress mRNAs containing abnormally expanded CAG repeats

Collaborative research on invasive epithelial cells in the precancerous state in interstitial pneumonia and the development of therapeutic agents

Anti-miRNA Anticancer Drugs

Improved S-TuD (Synthetic Tough Decoy) RNA nucleic acid that inhibits miR-200 family, which is highly expressed in mammary cancer, colon cancer, endometrial cancer, ovarian cancer, etc.

Bacteria derived membrane vesicles for delivery of antibiotics and nucleic acids

Using vesicles derived from the cell membrane of target bacteria allows for highly species-specific treatment

Novel Drug Target for Endometriosis

Identification of prostaglandin receptors involved in the pathogenesis of endometriosis. [Collaborative Research Proposal]

Upregulation of circular RNA as novel therapeutic concept

RNA-binding molecules, such as antisense oligonucleotides and small molecules, facilitate back-splicing progression and augment circular RNA

Polycystic Kidney Disease Treatment Drug

Therapeutic target molecules and antisense oligonucleic acid (ASO) therapeutic agents for autosomal dominant polycystic kidney disease (ADPKD).

Therapeutic Drugs for α-Synucleinopathy

Splice-switching antisense oligonucleotides (SSOs) targeting the α-synuclein (SNCA) gene suppress α-synuclein (α-Syn) aggregation.

IND Ready Nucleic Acid Medicine against Cancer Peritoneal Metastasis

ASO-4733: Optimized Anti-SYT13 antisense oligonucleotides

Lipids with novel cell-penetrating peptide for functionalized drug delivery system

Newly designed KK-(EK)4-lipid having near neutral charge enhances intracellular translocation of drug carrier

NF-κB inducing kinase (NIK) Novel target for cholangiocarcinoma

Molecular targeted drugs targeting the NIK associated with the differentiation factor from hepatocytes to cholangiocarcinoma

Prediction of the Efficacy of Cancer Immunotherapy by Direct Measurement of T-cell Cancer Cytotoxic Activity.

Also useful as a companion diagnostics (CDx) for immune checkpoint inhibitors (ICIs).

Improving the efficiency of exogenous mRNA expression

Overcoming the challenges of mRNA medicine by improving translation efficiency