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Table 1. Examples of RNA-based therapeutics in medicine
Drug FDA Clinical
Type of drug Active moiety Target Disease Reference
name approval studies
Antisense Fomivirsen Yes Completed 21-mer IE2 mRNA Cytomegalovirus [124]
oligonucleotides phosphorothioate
oligonucleotide
Pegaptanib Yes Completed 28-mer RNA aptamer VEGF165 Age-related macular [131]
protein degeneration
Nusinersen Yes Completed 18-mer 2′-O- ISS-N1 protein Spinal muscular atrophy [132]
methoxyethyl aptamer
Mipomersen Yes Completed 20-mer ApoB-100 Familial hypercholesterolemia [125]
phosphorothioate mRNA
oligonucleotide with 2′
-O-methoxyethyl
modification
Small interfering Patisiran Yes Completed 21-bp double-stranded Transthyretin Hereditary transthyretin [133]
RNAs siRNA encapsulated in mRNA amyloidosis
a lipid nanoparticle
Givosiran Yes Completed GalNAc-conjugated ALAS1 mRNA Acute hepatic porphyria [126]
21-bp double-stranded
siRNA
Lumasiran Yes Completed GalNAc-conjugated HAO1 mRNA Primary hyperoxaluria type 1 [134]
21-bp double-stranded
siRNA
Short hairpin N/A No Not yet Single-stranded SOD1 mRNA Amyotrophic lateral sclerosis [135]
RNAs shRNA on a scAAV
construct
MicroRNAs MRX34 No Halted miRNA-34a mimic in mRNA targets A variety of malignant or [130]
liposomal of miR-34a carcinogenic conditions,
nanoparticles including hepatocellular
carcinoma, ovarian cancer, colon
cancer, non-small cell lung
carcinoma, and cervical cancer
Miravirsen No Phase II AntimiR against miR-122 Hepatitis C [127]
miR-122
Remlarsen No Phase II miR-29b mimic mRNA targets Cutaneous and pulmonary [128]
of miR-29b fibrosis
RG-125 No Phase I/II AntimiR against miR-103/107 Non-alcoholic fatty liver disease [129]
miR-103/107

IE2: Immediate-early region 2 protein; VEGF: vascular endothelial growth factor; ISS-N1: intronic splicing silencer N1; ALAS1: δ-aminolevulinic acid
synthase 1; GalNAc: N-acetylgalactosamine; scAAV: self-complementary adeno-associated virus; SOD1: superoxide dismutase 1; HAO1:
hydroxyacid oxidase 1.

Structural and molecular diversity
LncRNAs can have several domains that are involved in different interactions; blocking these interactions
could have therapeutic value. Understanding the pertinent RNA motifs and their structural complexity is
necessary for finding ligands that can bind lncRNAs with high specificity and affinity. Currently, this level
of knowledge is only attainable for a small number of lncRNAs. The progress in this field will increase as
[121]
more information about lncRNAs' structural and molecular features becomes available .
Immunogenicity and tolerability
As a defense mechanism against viruses, our immune system identifies both single-stranded and double-
stranded RNA through various extracellular and intracellular pathogen-associated molecular pattern
[140]
receptors, including Toll-like receptors, which can trigger diverse immune responses . Additionally,
research has demonstrated that single-stranded RNA is more likely to induce immune stimulation
compared to double-stranded RNA. Consequently, all currently used or under-development siRNAs in
clinical applications are designed as double-stranded structures [106,141] .