1:30 ABO-101, a novel gene editing therapy for primary hyperoxaluria type 1, is efficacious and well tolerated in NHPs and results in high fidelity editing in primary hepatocytes
Time: 1:30 pm
day: Conference Day 2
Details:
- Single administration of ABO-101 to NHPs results in efficient HAO1 editing, reduced GO enzyme activity, and no clinical signs or adverse events
- mABO-101, a mouse surrogate of ABO-101, demonstrated efficient in vivo editing and reduction of urinary oxalate in juvenile and adult Agxt KO mice
- ABO-101 is an efficient and specific HAO1 gene editor in vitro with a favorable off-target profile
