After more than 20 years of ups and downs, the era of RNAi therapy finally here.
On January 7, 2021, Alnylam, a leading RNAi therapy research and development company, announced positive results from its Phase III trials on its RNAi therapy Vutrisiran Alnylam plans to submit the complete data package to the FDA in early 2021 and a new drug listing application (NDA). Vutrisiran, like the first RNAi drug Patisiran (intravenous preparation) (also developed by Alnylam), is used to treat neurological damage caused by hereditary thyroxine amyloidosis (hATTR). After its approval, Vutrisiran will complement Patisiran.
Figure 1: Silencing TTR gene expression can potentially address the underlying cause of disease.
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Thyroxine amyloidosis (ATTR) is a rare, progressive, and fatal disease that causes systemic failure due to the deposition of insoluble substances.
Figure 2: Mechanism of amyloid formation (Morie A. Gertz, 2019).
Vutrisiran is a subcutaneously injected RNAi therapy that uses enhanced stability chemistry (ESC)-GalNAc delivery technology to reduce target gene expression and block the production of wrong proteins by mediating target mRNA degradation and silencing mRNA translation, leaading to the inhibition of hATTR disease progression. Compared with Patisiran, the administration of Vutrisiran is more convenient. Patients undergoing experimental treatment receive a subcutaneous injection every three months.
Once Vutrisiran is approved, RNAi technology will become a reproducible drug platform to generate, innovative drugs for the treatment of rare genetic diseases, cardiometabolic diseases, liver infections, and central nervous system (CNS)/eye diseases.
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| siRNA Drug | Approved | Indication | Delivery Strategy |
| Patisiran (Onpattro) | 2018 | Hereditary thyroxine-mediated amyloidosis (hATTR) | Lipid nanoparticles (LNP) |
| Givlaari (givosiran) | 2019 | Acute hepatic porphyria (AHP) | GalNAc-siRNA conjugation |
| Oxlumo (lumasiran) | 2020 | Primary hyperoxaluria type 1 (PH1) | GalNAc-siRNA conjugation |
| Vutrisiran | 2021 | Hereditary thyroxine amyloidosis (hATTR) | GalNAc-siRNA conjugation |
RNAi uses small interfering RNA (siRNA) to specifically bind target mRNA, triggering degradation and preventing the production of corresponding proteins, enabling precise control over gene expression.
Enhanced delivery systems such as GalNAc conjugates, lipid nanoparticles, and polymer carriers improve cellular uptake, tissue targeting, and stability of RNAi molecules in experimental studies.
RNAi allows researchers to selectively reduce expression of mutant genes, helping to explore protein function, disease pathways, and gene regulation in models of inherited disorders.
Challenges include achieving efficient cellular uptake, avoiding off-target effects, maintaining siRNA stability, and ensuring effective engagement of the RNAi machinery within target cells.
Yes, RNAi technology is versatile and can be applied in gene function studies, protein regulation research, infectious agent modeling, and cardiometabolic or neurological experimental systems.
Chemical modifications such as ESC or conjugation with GalNAc improve RNAi stability, targeting specificity, and efficiency of gene silencing, supporting reproducible and scalable experimental applications.
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