Science & Pipeline

A Robust Drug Discovery Platform Has Been Established
to Develop Novel, Potent and Safe RNAi Therapeutics

RNAi Mechanism

Since the discovery of RNAi was awarded for the Nobel Prize of 2006 in Physiology or Medicine, 6 drugs were approved by FDA in the past years, demonstrating that RNAi is an effective novel drug modality with great advantages over traditional drugs.

RNAi, in a form of short oligonucleotides, triggers the gene silencing to reduce the protein production by leveraging an evolutionarily conserved mechanism mediated by an RNA-induced silencing complex (RISC). This mechanism offers sequence-based high specificity and remarkable targetability to disease-causing genes. Sanegene' s RNAi-based therapeutics have achieved effective and durable impacts to silence the target genes specifically in different organs.


RNAi Therapeutics

RNA molecules exhibit unique properties, such as highly charged backbone and limited membrane permeability. These traits have constrained its widespread application as a drug modality for diverse disease indications. A breakthrough is needed to surmount these barriers, deliver RNAi molecules to target organs while maintaining a desired pharmacodynamic/pharmacokinetic profile, and unlock the full potential of RNAi therapeutics in a broad range of disease areas.


SanegeneBio has established a robust siRNA drug discovery platform, LEAD™ (Ligand and Enhancer Assisted Delivery), reinforced by a proprietary modular design concept encompassing tissue-specific targeting ligands, delivery enhancers, and optimized chemical modifications. Our LEAD™ platform enables the efficient design, screening, identification, and development of new RNAi molecules with enhanced tissue distribution and improved therapeutic and safety profiles. This innovation extends the reach of RNAi medicines beyond hepatic tissues, addressing diverse extrahepatic disease targets and unleashing the full potential of RNAi technologies in areas with unmet medical needs.

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