Drug intelligence / Profile preview

AIC468

Development stage
Unknown
Lead developer
AiCuris
Modality
Small Molecules, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous, Subcutaneous (preclinical Data)
01

Overview

AIC468 is a novel antisense oligonucleotide (ASO) antiviral agent developed to target BK virus (BKV) infections, particularly in kidney transplant recipients. BKV reactivation in immunocompromised patients, such as those undergoing organ or bone marrow transplantation, can lead to serious complications including graft loss and BK virus-associated nephropathy. There are currently no approved antiviral treatments specifically for BKV. AIC468 works by binding to specific RNA sequences of the BK virus, thereby inhibiting viral replication at the molecular level without compromising ongoing immunosuppressive therapy. Preclinical studies have demonstrated that AIC468 achieves efficacious concentrations in kidney tissue and effectively reduces BKV replication in human kidney cells[1][2][7][8]. The drug is currently being evaluated for safety, tolerability, and pharmacokinetics in healthy volunteers as part of a Phase 1 clinical trial[2][5][6].

02

Targets

BK polyomavirus early region pre-mRNA splice donor site

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