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, 4 drugs were approved by FDA in the past 5 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

Certain properties of RNAi such as being highly charged and unfavorable membrane permeability have limited its full potential as a drug modality to apply to a broad range of disease indications. Breakthrough is needed to overcome different barriers to realize an optimal pharmacologic activity for RNAi therapeutics in different targeted organs.


SanegeneBio has established a robust drug discovery platform based on the proprietary modular design of payload and conjugation. The platform will further enable the company to develop novel, potent and safe therapies in a rapid and cost-effective manner. Sanengene's platform is consisted of a RNAi payload with optimized compositions including potent sequences, enhanced and stabilized chemical modifications and minimized off-target features, together with a high affinity targeting ligand to further improve pharmacokinetics and pharmacodynamics for a strong RNAi drug candidate. Sanegene's platform will unleash the true potential of RNAi therapeutics by targeting extrahepatic tissues, reaching multiple organs including CNS, muscles, and adipose to fill the gap for the unmet medical needs in these areas.

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