Arbor sails into rocky gene editing waters with $74M for rare liver disease therapy
Arbor Biotechnologies raised $73.9 million in Series C financing to advance its gene editing therapies.
The funding will support ABO-101, a CRISPR-based gene editing therapy for primary hyperoxaluria type 1 (PH1), a rare liver disease.
ABO-101 uses Arbor's Type V CRISPR Cas12i2 nuclease to target the HAO1 gene in the liver.
It's designed as a one-time liver-directed gene editing treatment to reduce PH1-associated oxalate production.
The FDA has granted ABO-101 Orphan Drug and Rare Pediatric Disease designations.
Arbor is advancing ABO-101 in the RedePHine Phase 1/2 clinical trial.
The company is also developing therapies for other rare liver diseases and amyotrophic lateral sclerosis (ALS).
This funding comes as other gene editing programs have faced challenges or been sold off recently.
Arbor aims to deliver novel gene editing therapeutics, including for CNS diseases with high unmet need.