Drug intelligence / Profile preview

Apta-1

Development stage
Unknown
Lead developer
Aptahem
Modality
RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

Apta-1 is a lead RNA-based aptamer therapeutic developed by Aptahem for the treatment of sepsis and other acute inflammatory conditions. It possesses a unique multifunctional profile characterized by anti-inflammatory, anti-thrombotic, and immunomodulating properties. Mechanistically, Apta-1 specifically recognizes and deactivates thrombin by binding to a site distinct from the enzyme's active site, which helps reduce uncontrolled blood clotting and systemic inflammation while potentially minimizing bleeding risks. Preclinical studies have shown its ability to inhibit inflammatory cytokines, reduce clot growth, and prevent tissue degradation. A Phase 1 first-in-human clinical trial was completed in June 2024, demonstrating that the drug is safe and well-tolerated.

02

Targets

F2 (Thrombin)

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