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Bacterial Delivery of RNAi Effectors: Transkingdom RNAi
Published on: August 18, 2010
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RNAi therapeutic and its innovative biotechnological evolution
Yuhua Weng1, Haihua Xiao2, Jinchao Zhang3
1Advanced Research Institute of Multidisciplinary Science, School of Life Science, Key Laboratory of Molecular Medicine and Biotherapy, Beijing Institute of Technology, Beijing 100081, PR China.
Biotechnology Advances
|April 30, 2019
Summary
The first RNA interference (RNAi) therapeutic, ONPATTRO™ (Patisiran), is now approved for hereditary transthyretin-mediated amyloidosis. This landmark approval signifies a breakthrough for RNAi drug development from research to clinical application.
Area of Science:
- Biotechnology
- Pharmacology
- Genetics
Background:
- Hereditary transthyretin-mediated (hATTR) amyloidosis is a progressive, multi-system disease.
- Previously, treatment options for hATTR amyloidosis were limited.
- RNA interference (RNAi) therapeutics offer a novel mechanism for gene silencing.
Purpose of the Study:
- To provide a comprehensive overview of ONPATTRO™ (Patisiran), the first approved RNAi therapeutic.
- To discuss the properties of RNAi and nucleic acid therapeutics.
- To review the development, technologies, and future prospects of RNAi therapy.
Main Methods:
- Review of clinical and preclinical data for ONPATTRO™.
- Analysis of RNAi technology, including early-stage key technologies.
- Examination of advancements in RNAi delivery and modification techniques.
Main Results:
- ONPATTRO™ (Patisiran) received approval from the FDA and EC for hATTR amyloidosis.
- This marks the first global approval of an RNAi therapeutic and the first FDA-approved treatment for this indication.
- The approval represents a significant milestone, validating the RNAi therapeutic pathway.
Conclusions:
- The approval of ONPATTRO™ is a pivotal moment for RNAi therapeutics, paving the way for future drug development.
- Continued advancements in delivery and modification technologies are crucial for the broader application of RNAi therapies.
- RNAi therapy holds significant promise for treating various genetic disorders.
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