On Aug. 10, 2018, the United States Food and Drug Administration (FDA) approved our licensor-- Alnylam’s ONPATTRO? (patisiran) as the first ever FDA-approved RNAi medicine. This historical approval marks the arrival of an entirely new class of therapeutics — RNAi Medicines.
RNAi is a naturally occurring cellular mechanism of regulating gene expression and is mediated by small interfering RNAs (siRNAs).
We aim to develop ASC06 as the first systematically delivered therapeutic drug to treat liver cancer by using RNA interference, a breakthrough approach to drug discovery and development. ASC06 has been designed to silence two genes critical for growth of liver cancer cells — vascular endothelial growth factor (“VEGF”) and kinesin spindle protein (“KSP”). ASC06 has completed phase I and phase I extension clinical trials, which have shown that 50% of patients who received ≥0.7 mg/kg dose achieved stable disease and one patient achieved a complete response. ASC06 is safe and well-tolerated by patients.
* Intracellular double stranded RNA is processed by the “dicer” complex pathway (A) to produce siRNAs which become integrated into a multi-subunit protein complex, the RNAi induced silencing complex (RISC) (B), which guides the siRNAs to the target mRNA sequence (C). The siRNA duplex unwinds, and the antisense strand remains bound to RISC and directs degradation of the complementary mRNA sequence (D), resulting in target protein suppression (E).