Drug intelligence / Profile preview

ASO-snoRNA

Development stage
Preclinical
Lead developer
Duke University
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous
01

Overview

ASO-snoRNA is an antisense oligonucleotide (ASO) therapy designed to target and reduce the levels of specific small nucleolar RNAs (snoRNAs) encoded within the introns of the *Rpl13a* gene, specifically U32a, U33, U34, and U35. These snoRNAs are known to elevate cellular levels of reactive oxygen species (ROS), which contribute to the progression of atherosclerosis. Developed by researchers at Duke University, ASO-snoRNA has demonstrated the ability to reduce atherosclerotic lesion area and circulating inflammatory markers (such as IL-1β) in preclinical mouse models (*Apoe-/-*). The therapy is administered via subcutaneous injection and aims to mitigate atherosclerosis in both early and late stages of the disease.

Other names
Rpl13a-snoRNA ASORpl-13a-snoRNA ASORpl 13a-snoRNA ASOantisense oligonucleotides targeting Rpl13a-snoRNAs
02

Targets

SNORD35A (Small nucleolar RNA, C/D box 35A)SNORD34 (Small nucleolar RNA, C/D box 34)SNORD33 (Small nucleolar RNA, C/D box 33)SNORD32A (Small nucleolar RNA, C/D box 32A)

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