Drug intelligence / Profile preview

APT-003S

Development stage
Unknown
Lead developer
Aptah Bio
Modality
RNA Therapeutics → Nucleic Acid Therapeutics
01

Overview

APT-003S is an investigational RNA therapy developed by Aptah Bio for the treatment of lung cancer. It utilizes the company's proprietary RNA WiCo™ platform to target the U1 small nuclear ribonucleoprotein (U1 snRNP), a key component of the spliceosome. By modulating U1 snRNP activity, APT-003S aims to regulate RNA splicing and processing, which are frequently dysregulated in oncogenic pathways. The drug is currently in the proof-of-concept stage of development, with studies reaching the Phase 1/2 level.

Other names
APT-003S-Aptah Bio-U1 snRNP-RNA WiCo™ (RNA therapy)-Lung Cancer
02

Targets

U1 snRNP (U1 small nuclear ribonucleoprotein)

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