Solutions by Modality

Gene Therapy

We conduct preclinical studies for gene therapies, utilizing various platform technologies such as AAV, pDNA, and mRNA. By analyzing the complex mechanisms of gene delivery technologies, we support successful IND submissions and regulatory approvals through tailored study designs.

Particularly, we excel in TK (toxicokinetics) and PK (pharmacokinetics) analysis for small oligonucleotide-based therapeutics such as ASOs (antisense oligonucleotides), collaborating with global ASO leaders to establish proprietary analytical methods.

Key Services

  • Efficacy evaluation: Analysis of gene expression levels and therapeutic effects
  • Immunogenicity studies: Assessment of immune responses and resistance
  • PK/distribution studies: Tissue specificity and in vivo stability evaluation
  • Safety and toxicity studies

Highlights

  • Expertise in diverse gene delivery technologies (AAV, LNP, pDNA, mRNA)
  • Proprietary analytical methods developed in collaboration with global ASO leaders
  • Extensive experience in domestic and international ASO and gene therapy projects