Yes, several RNA-based therapeutics using GalNAc conjugation have been approved for clinical use, particularly for liver-targeted therapies. Some of the approved drugs that utilize GalNAc for targeted delivery include:
● Patisiran (siRNA): Targets transthyretin (TTR) to treat hereditary transthyretin-mediated amyloidosis.
● Approval Year: 2018
● Regulatory Body: FDA
● GalNAc Use: Patisiran uses GalNAc conjugation to enhance liver-specific delivery of the siRNA.
● Givosiran (siRNA): Targets aminolevulinate synthase 1 (ALAS1) to treat acute hepatic porphyria.
● Approval Year: 2019
● Regulatory Body: FDA
● GalNAc Use: Givosiran also utilizes GalNAc conjugation to target delivery to liver cells.
● Vupanorsen (ASO): Aiming to reduce apoC-III for treating hypertriglyceridemia.
● Approval Year: 2024
● Regulatory Body: EMA (European Medicines Agency)
● GalNAc Use: Vupanorsen uses GalNAc for liver-targeted delivery of antisense oligonucleotides.
These drugs demonstrate how GalNAc conjugation is effectively used to enhance the delivery and therapeutic impact of small nucleic acids, particularly in liver-targeted treatments.
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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