RNA Technology

Oligonucleotide therapeutics has seen a rapid growth following several recent clinical successes.  As of 2021, 13 oligonucleotide therapies including nine antisense oligonucleotides (ASOs) and four small interfering RNAs (siRNAs) have been approved by the FDA for commercial use for a variety of diseases.  Many more are currently in clinical trials. [1] 

 

ASO therapeutics are short DNA-like molecules that bind to cellular RNA targets and modify their expression by either knocking them down, increasing expression or by modulating RNA splicing.  For example, one highly efficacious and safe splicing drug called Spinraza was approved by the FDA in 2016 to treat spinal muscular atrophy (SMA).   

 

These advances in ASO technology, next generation sequencing and new tools for statistical computing have allowed La Jolla Labs to design and screen for highly specific and personalized therapies to treat rare and ultra-rare genetic diseases.

 

La Jolla Labs is paving the way for cost-effective rare disease and personalized therapeutics. 

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1. Crooke ST, Baker BF, Crooke RM, Liang XH. Antisense technology: an overview and prospectus. Nat Rev Drug Discov. 2021 Jun;20(6):427-453. doi: 10.1038/s41573-021-00162-z. Epub 2021 Mar 24. PMID: 33762737.