RNA Synthesis & Modifications—Approved small nucleic acid drugs

What are the approved small nucleic acid drugs currently on the market?

Small nucleic acid drugs have gained regulatory approval for the treatment of various diseases, showcasing their therapeutic potential. Below are examples of these approved drugs:

Drug	Type	Target/Disease	Approval Year	Regulatory Body
Patisiran	siRNA	Transthyretin (TTR) / Hereditary amyloidosis	2018	FDA
Givosiran	siRNA	ALAS1 / Acute hepatic porphyria	2019	FDA
Nusinersen	ASO	SMN2 / Spinal muscular atrophy (SMA)	2016	FDA
Eteplirsen	ASO	DMD gene / Duchenne muscular dystrophy (DMD)	2016	FDA
Mipomersen	ASO	ApoB-100 / Homozygous familial hypercholesterolemia	2013	FDA
Pegaptanib	Aptamer	VEGF / Age-related macular degeneration (AMD)	2004	FDA

Approved small nucleic acid drugs demonstrate efficacy in genetic disorders, metabolic diseases, and ophthalmic conditions, highlighting their diverse therapeutic applications.

GenCefe Biotech provides high-quality siRNA, miRNA, sgRNA, and custom RNA synthesis services. We can design and synthesize RNA Oligos of different lengths, different forms, and with various modifications according to customer’s needs, and also synthesize RNA sequences designed by customers. The RNA products we deliver are all purified by HPLC. ISO 9001-certified facilities and comprehensive quality control reports ensure the delivery of high-quality products.

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