Advancing Antisense Oligonucleotide Therapies for Rare Diseases

Authors

• - Atena Farkhondeh Kalat
• - William Borschel1
• - Elia R Lopez
• - Jyoti Roy
• - Edward Wu
• - Elizabeth A Ottinger
• - Bryan J Traynor

Abstract

Antisense oligonucleotides (ASOs) are short, synthetic, single-stranded oligonucleotides that can bind RNA and selectively alter gene expression, heralding a new era of precision medicine. This therapeutic approach has been used successfully to treat rare diseases, such as spinal muscular atrophy and Duchenne Muscular Dystrophy. ASO technology continues to evolve and improve, ultimately providing effective, safe therapies for a range of diseases. NCATS’ goal is to bring more treatments to all people more quickly. At the Therapeutic Development Branch in NCATS’ intramural Division of Preclinical Innovation, we have been developing a platform using cell culture models to select RNA-directed gene therapies candidates and predict toxicity to further enable ASO therapeutic development for rare and ultra-rare diseases. Our portfolio activities are focused on (i) lead identification/optimization of ASOs to select candidates for further therapeutic development and enable eventual clinical trials in patients with rare and neglected diseases, (ii) improved prediction of potential ASO toxicity using in vitro assays, and (iii) safer delivery of ASOs to the CNS using lipid nanoparticles.

Scientific Focus Area: Molecular Pharmacology

This page was last updated on Tuesday, August 6, 2024