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We create oligonucleotide candidates by one-stop drug discovery service.

Our oligonucleotide drug (OND) discovery

1.Creating OND candidates by using solution technology, data, and infrastructure

2.Assessments and support for research and development of ONDs toxicities

3.Solutions to challenges for OND’ s delivery to target tissues and cells

Design of sequences and synthesis

Efficient optimization of antisense oligonucleotide (ASO) sequences for target mRNAs.

Evaluation of pharmacokinetic and localization in tissue and cell

One possible cause of insufficient drug efficacy is poor delivery to cells in the target tissue. We can execute quantitative analysis and visualization by immunohistochemistry and in-situ hybridization to evaluate distribution   of oligonucleotides at the cellular level.

Quantitative analysis

Based on our various experiences, we choose the best method, such as LC-MS/MS and Hybridization ELISA and report the results promptly. In addition, we can also perform analysis of ligand conjugate oligonucleotides and metabolites.

Visualization by immunohistochemistry (IHC) and in situ hybridization (ISH)

We analyze delivery and activity at the cellular level using co-staining of ASO by IHC using anti-PS antibodies and target mRNA by ISH. It is also possible to stain the administered ASO with ISH.

Delivery of therapeutic oligonucleotides

To address the issue of OND delivery, we comprehensively support approaches that utilize peptide-oligonucleotide conjugates and lipid nanoparticles (LNPs).

Design of peptide-oligonucleotide conjugates (POC)

Peptide conjugation is an effective approach to deliver oligonucleotides to the site of action. Design and synthesis of appropriate peptide-oligonucleotide conjugates can solve challenges of delivery to targeted tissues and cells. Our extensive experience in peptide drug discovery allows us to develop peptide-nucleotide conjugates tailored to your requirements.

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LNP formulation and biodistribution assessment

Axcelead support development of in vitro/in vivo platforms customized for a client for new modalities.

Assessment of drug efficacy evaluation

・Various phenotypic methods using human cells (disease models)

Axcelead prepares a variety of human cells (disease models), using various cell lines and hiPS cell-derived differentiated cells, and conducts various phenotypic analyses in order to select or prioritize OND candidates for our clients.

・Assessment of drug efficacy using animal disease models

The efficacy (PD/drug efficacy) of OND candidates for animal disease models with high clinical extrapolation capability is assessed and data for a clinical trial are provided.

・Generation of genetically-modified mice and rats

Using genome-editing technology (CRISPR/Cas9 system), genetically-modified mice and rats, with a targeted gene deleted or mutation inserted, can be efficiently generated and provided.

Assessment of drug toxicities

We have a variety of evaluation systems that cover a wide range of toxicities in ONDs, helping to design and select less toxic and more effective oligonucleotides.