The ASO for myotonic dystrophy type 1 was well tolerated and improved splicing and function in a study presented at ASGCT.

In higher eukaryotes, a vast variety of RNAs is transcribed from genomic DNAs. The transcribed RNAs are subjected to RNA processing steps, such as splicing, ...

QurAlis' FlexASO® platform was developed to generate splice-switching ASOs with improved potency, increased therapeutic index and improved bio-distribution. This bespoke platform has the potential to ...