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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.
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