1. Assessment of the delivery to cells in target tissues (PK/PD analysis)
One possible cause of insufficient drug efficacy is poor delivery to cells in the target tissue.
Axcelead can address challenges in delivery by combining various methods and identify processes that lead to bottlenecks.
Based on the biophase PK and PD analysis results, it is firstly determined whether delivery can provide sufficient efficacy. In addition, dosing regimes for a pharmacological study can also be proposed.
Assessment of drug efficiency using animal disease models
Assessment of plasma/organ concentrations
Rapid assessment of plasma and organ concentrations using state-of-the-art equipment
Owned LC/MS Instrument
・ Orbitrap Exploris 120
Assessment of intracellular localization
Assessment of intracellular behavior (e.g., endosomal escape) using fluorescence-labeled oligonucleotides and organelle markers
Red: Oligonucleotide escaped from the endosome
Yellow: Oligonucleotide remaining in the lysosome
2. Design and synthesis of peptide-oligonucleotide conjugates
One solution to address challenges in delivery by nucleic acid drugs is the use of peptides. To overcome challenges in delivery to target tissues and cells, appropriate peptide-oligonucleotide conjugates are designed and synthesized.
▶Synthesis of cell-penetrating peptides and target receptor-binding peptides
▶Proposal of the optimal linker according to the characteristics of the peptides
▶Synthesis and purification of conjugates
Axcelead offers peptide-oligonucleotide conjugates that are tailored to our clients’ objectives by taking full advantage of our “infrastructure, technology and know-how” cultivated in the field of peptide drug discovery.
3.LNP formation and in vivo biodistribution analysis
Axcelead support development of in vitro/in vivo platforms customized for a client for new modalities.