ReviewBiochemical pharmacology2021
The growth of siRNA-based therapeutics: Updated clinical studies.
Review in Biochemical pharmacology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 252 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
252 citing papers in PubMed, 1 synthesis or guideline pooled it, 471 citations in OpenAlex.
- Safety profile of small interfering RNA therapeutics: a systematic review and meta-analysis of randomized controlled trials.Frontiers in drug safety and regulation · 2026Pooled it
- A Novel Injectable Polypeptide Nanoparticle Encapsulated siRNA Targeting TGF-β1 and COX-2 for Localized Fat Reduction II: Phase I Clinical Trial.Journal of cosmetic dermatology · 2025Trial
- SPI1-driven POSTN transcription promotes esophageal cancer progression and macrophage immunosuppressive polarization.Histology and histopathology · 2026Article
- RNA therapeutics: current status and future directions.Signal transduction and targeted therapy · 2026Review
- RNAi-based therapeutics targeting the F gene in human metapneumovirus: An in-silico approach.Journal, genetic engineering & biotechnology · 2026Article
- Polymeric mesoscale nanoparticles exhibit dynamin- and macropinocytosis-dependent endocytosis.Nanomedicine : nanotechnology, biology, and medicine · 2026Article
- Article
- Zilebesiran, a Small Interfering RNA Therapeutic Targeting Angiotensinogen: Mechanism, Clinical Evidence, Safety, and Implementation Considerations.Life (Basel, Switzerland) · 2026Review
- Polyplex of peptide-mannan and RNA for intranasal delivery of TGF-β siRNA in treatment of pulmonary fibrosis.Bioactive materials · 2026Article
- Therapeutic siRNA: Mechanisms, challenges, strategies, and clinical translation.Chinese medical journal · 2026Review
- Hsa-miR-25-3p Inhibition Sensitizes Patient-Derived Glioblastoma Cells to Temozolomide via β-catenin Downregulation.Cellular and molecular neurobiology · 2026Article
- Optimizing Oligonucleotide Therapeutics: A Model-Informed Drug Development Perspective.Clinical and translational science · 2026Review
- Amidation of Phosphonoacetate-Modified Oligonucleotides and Evaluation of the Properties of the Synthesized Oligonucleotides.Organic letters · 2026Article
- The application of nanoparticle-mediated siRNA delivery systems in ovarian cancer.Cancer gene therapy · 2026Review
- Preparation of SP94-Modified Calcium Phosphate Lipid Nanoparticles Loaded with Bcl-2 siRNA and Doxorubicin and Their Targeted Therapeutic Effect on Hepatocellular Carcinoma.Pharmaceutical research · 2026Article
- The PAH-AM-PEG-ApoE@siRNA Nanocarrier Delivery System for Polo-like Kinase 1 Inhibition Suppresses Glioma Progression.AAPS PharmSciTech · 2026Article
- Endocytosis of PEGylated polymeric mesoscale nanoparticles is dynamin- and macropinocytosis-dependent.bioRxiv : the preprint server for biology · 2026Article
- The Molecular Mechanisms and Therapeutic Potentials of Engrailed-1 in Fibrotic Scar Formation Post Glaucoma Filtration Surgery.Investigative ophthalmology & visual science · 2026Article
- Cholesterol-dependent control of endosomal escape regulates intracellular trafficking of small interfering RNA therapeutics and interactions with small molecule drugs.The Journal of pharmacology and experimental therapeutics · 2026Article
- Nanotherapeutic potential in glaucoma associated ocular cancers.Discover oncology · 2026Review
192 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 1 institution in 1 country.
Funding
Abstract
More than two decades after the natural gene-silencing mechanism of RNA interference was elucidated, small interfering RNA (siRNA)-based therapeutics have finally broken into the pharmaceutical market. With three agents already approved and many others in advanced stages of the drug development pipeline, siRNA drugs are on their way to becoming a standard modality of pharmacotherapy. The majority of late-stage candidates are indicated for rare or orphan diseases, whose patients have an urgent need for novel and effective therapies. Additionally, there are agents that have the potential to meet the need of a broader population. Inclisiran, for instance, is being developed for hypercholesterolemia and has shown benefit in patients who are uncontrolled even after maximal statin therapy. This review provides a brief overview of mechanisms of siRNA action, physiological barriers to its delivery and activity, and the most common chemical modifications and delivery platforms used to overcome these barriers. Furthermore, this review presents comprehensive profiles of the three approved siRNA drugs (patisiran, givosiran, and lumasiran) and the seven other siRNA candidates in Phase 3 clinical trials (vutrisiran, nedosiran, inclisiran, fitusiran, teprasiran, cosdosiran, and tivanisiran), summarizing their modifications and delivery strategies, disease-specific mechanisms of action, updated clinical trial status, and future outlooks.
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Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.