Topics
Dr. Takakuwa from Translational Research Business Unit to present a poster at the 11th Annual Meeting of Nucleic Acids Therapeutics Society of Japan
2026.06.29
Dr. Takakuwa, a researcher from the Translational Research Business Unit, will have a poster presentation at the 11th Annual Meeting of Nucleic Acids Therapeutics Society of Japan, to be held at Dejima Messe Nagasaki, Japan.
If you plan to attend the conference, we encourage you to join the session and hear the presentation.
Conference Name: the 11th Annual Meeting of Nucleic Acids Therapeutics Society of Japan
Date & Time: July 8 (Wed), 2026, Poster Discussion: 11:10–12:10
Location: Dejima Messe Nagasaki, Japan
Title: Pharmacological and Toxicological Profiling and Efficacy Predictability of an Antisense Oligonucleotide Following Repeated Intrathecal Administration
Poster Board No: P090
Presentation Summary
Because oligonucleotide therapeutics have limited blood–brain barrier permeability, direct CNS administration routes such as intracerebroventricular (ICV) and intrathecal (IT) injection are commonly used. Previous studies have shown that ASO efficacy and toxicity are influenced by the route of administration and do not necessarily change in parallel.
In this presentation, we evaluate the relationships among ASO exposure in plasma, cerebrospinal fluid (CSF), and tissues, tissue target knockdown (KD), and acute CNS toxicity following IT administration. Our findings suggest that distinct ASO distribution patterns may underlie KD activity and acute CNS toxicity, providing practical insights for the design of nonclinical studies and the selection of dosing regimens for CNS-targeting ASOs.
Axcelead’s Soulution
At Axcelead, we have established an integrated evaluation platform that combines our CNS direct delivery technology and nucleic acid analytics. We provide one-stop services from sequence design to efficacy and safety assessment. Please feel free to contact us when considering the evaluation of CNS-targeted nucleic acid therapeutics.
Related Service: Oligonucleotide
Misae Takakuwa Ph.D. Translational Research Business Unit
Has over 15 years of experience in central nervous system (CNS) pharmacology within the pharmaceutical company.
Joined Axcelead Drug Discovery Partners, Inc. in 2023. Since joining, has been involved in nucleic acid drug discovery programs targeting CNS disorders, in addition to broader drug discovery research focused on psychiatric and neurodegenerative diseases.