X-linked Alport syndrome is an inherited kidney disease caused by pathogenic mutations in the COL4A5 gene. Patients develop hematuria, proteinuria and kidney function decline leading to end-stage renal disease. Nionyx Bio Inc. has developed ONYX-101, a novel kidney-targeting therapeutic designed to ensure durable COL4A5 restoration through dual-vector AAV delivery using NYX capsids that were optimized for kidney targeting.
Circio Holding ASA and Aavigen GmbH have entered a research collaboration focused on developing circVec-enhanced AAV vectors engineered for targeted, low-dose gene therapies for heart diseases.
New regulations tighten regulatory oversight of China’s investigator-initiated trials (IITs) but legitimize the pathway that will be open to other modalities beyond cell and gene therapies.
