Dan Elbaum
Chief Scientific Officer QurAlis
Seminars
Friday 21st August 2026
The Discovery & Early Development of QRL-201: A Splice Switching ASO Targeting STMN2 for the Treatment of ALS
11:00 am
- ALS is characterized by the mis-splicing of RNA resulting from TDP-43 LOF
- STMN2 pre-mRNA mis-splicing leads to loss of neuromuscular junctions and motor dysfunction
- The discovery and early development of QRL-201, a splice switching ASO that corrects the mis-splicing of STMN2, will be presented
