Pipeline Overview
Each program applies the same biological framework, with sequence design optimized for the survival gene context of each cancer type.
NUAgo is translating its RNA-based platform into multiple oncology programs. Program selection is guided by three criteria: sequence optimization for the biology of each tumor type, significant unmet medical need, and validated systemic delivery approaches.
This strategy reduces development risk and supports a more efficient path to clinical evaluation and potential commercialization.
The company’s lead program, NU003, targets hepatocellular carcinoma and serves as the initial clinical entry point for the platform. Additional programs are advancing in parallel to extend the platform across multiple tumor types.
NU003 — Hepatocellular Carcinoma
NU003 is an optimized short RNA therapeutic for hepatocellular carcinoma (HCC), one of the most common and deadly forms of liver cancer worldwide. HCC accounts for more than 80% of primary liver cancers and causes approximately 700,000 deaths each year, with the highest burden in Asia.
HCC presents significant therapeutic challenges due to tumor heterogeneity, treatment resistance, and limited effective treatment options. NU003 is designed to address these challenges by disrupting survival gene networks required for cancer cell viability.
The therapeutic sequence has been optimized to engage survival gene expression patterns associated with liver tumors and is paired with validated systemic delivery approaches appropriate for hepatic targeting. The liver is one of the most established organs for systemic RNA delivery, providing a practical entry point for translating the platform into clinical development.
NU003 represents the company’s lead development program and the initial clinical entry point for the platform, advancing a therapeutic candidate designed to translate the platform into a partner-ready oncology asset.
NU001 — Ovarian Cancer (NIH Funding, Combined Phase I/II STTR Grant)
NU001 applies the platform to ovarian cancer, a disease with substantial global mortality. More than 300,000 women are diagnosed with ovarian cancer each year and over 200,000 deaths occur annually worldwide.
Although PARP inhibitors have improved progression-free survival for some patients, treatment resistance and disease recurrence remain common after repeated therapy.
NU001 uses optimized short RNA sequences designed for ovarian tumor biology to disrupt survival gene networks and engage the conserved cell death mechanism underlying the NUAgo platform.
NU002 — Prostate Cancer (NIH Phase I STTR Grant)
NU002 is a platform-derived therapeutic program targeting prostate cancer, one of the most commonly diagnosed cancers in men worldwide.
The program applies optimized short RNA sequences designed to disrupt survival gene networks associated with prostate tumor biology.
NU002 is supported by a Phase I STTR grant from the National Institutes of Health.