Table 1.

FDA-approved RNA therapeutics for the treatment of human diseases in chronological order, adapted from [21,22].

Drug Name	Drug Class	Brand Name	Company	Target Disease	Mechanism of Action	Year of Approval	Current Status
Fomivirsen	ASO	Vitravene	Novartis	Cytomegalovirus retinitis	Binds to and blocks translation of IE2 mRNA.	1998	Withdrawn due to decreased need
Pegaptanib	Aptamer	Macugen	OSI Pharmaceuticals	Age-related macular degeneration (wet type)	Binds to and blocks the 165 isoform of VEGF.	2004	Continuous
Mipomersen	ASO	Kynamro	Genzyme Corporation	Homozygous familial hypercholesterolemia	Binds to ApoB mRNA and induces its degradation by RNase H.	2013	Discontinued due to side effects
Nusinersen	ASO	Spinraza	Cold Spring Harbor Laboratory and Ionis Pharmaceuticals	Spinal muscular atrophy	Binds to SMN2 mRNA and alters its splicing.	2016	Continuous
Eteplirsen	ASO	Exondys 51	Sarepta Therapeutics, Inc.	Duchenne muscular dystrophy	Binds to exon 51 and alters splicing of dystrophin pre-mRNA.	2016	Continuous
Patisiran	siRNA	Onpattro	Alnylam Pharmaceuticals Inc.	Polyneuropathy in patients with hereditary transthyretin-mediated amyloidosis.	Binds to transthyretin (TTR) mRNA to decrease hepatic production of TTR protein	2018	Continuous
Inotersen	ASO	Tegsedi	Ionis Pharmaceuticals	Nerve damage in adults with hereditary transthyretin-mediated amyloidosis.	Binds to TTR mRNA and induces its degradation by RNase H	2018	Continuous
Givosiran	siRNA	Givlaari	Alnylam Pharmaceuticals Inc.	Acute hepatic porphyria	Reduces the hepatic production of ALASI protein through interference with ALASI mRNA.	2019	Continuous
Golodirsen	ASO	Vyondys	Sarepta Therapeutics, Inc.	Duchenne muscular dystrophy	Binds to exon 53 of dystrophin pre-mRNA to alter splicing.	2019	Continuous

Note: Antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs).