Spotting Stubborn Indirect Effects in Oligonucleotide Therapeutics

Time: 4pm CET
Date: March 16, 2023

We use chemistry to improve the properties of nucleic acid therapeutics including antisense oligonucleotides, siRNAs, and genome editing approaches.

I will walk through a recent study we carried out trying to activate frataxin expression to treat Friedreich’s Ataxia.  We did a lot of the traditional “right” controls but still found ourselves vulnerable to off-target effects.  Finally, a combination of next-gen sequencing and gene editing proved that our effect was indirect.