ASO Therapies: Rapidly Evolving RNA-Based Therapeutics
Antisense oligonucleotide (ASO) technology allows targeted modulation of gene expression via short nucleotide sequences. Advances in ASO chemistry have improved stability, binding affinity, and tissue specificity, expanding their potential applications.
From Nusinersen, the FDA-approved 2016 drug for Spinal Muscular Dystrophy (SMD), to Tofersen, designed for SOD1-ALS and FDA-approved in 2023, multiple ASO approvals highlight their therapeutic potential in addressing specific genetic mutations linked to neurodegenerative disorders, paving the way for personalized treatments.
Antisense Oligonucleotide-based drugs are one of the promising therapeutic agents for the treatment of various human diseases. However, several issues must be overcome in the development of ASO based drugs such as:
Generations of ASO Therapies
Revolutionizing ASO therapy with AI and innovative approaches
In silico tools aid in providing a better understanding of available genomic and transcriptomic data, which includes sequence, structure, expression, and function.
Aganitha’s Point of View
We utilize Generative AI and ML in Antisense Oligonucleotide therapy to enhance target design, and optimize treatment.
Predicting the most effective sequences for targeting specific RNA molecules, and analyzing the structural and sequence properties of the target RNA to optimize ASO design for enhanced binding affinity and specificity.
Collating omics data, such as evolutionary scores, biomarkers, and regulatory elements, from varied authentic sources to facilitate target identification.