Also known as: AAV
A review in Genes & Diseases says robust non-clinical safety assessment is critical for CRISPR-based gene therapies. It highlights genotoxic, delivery, and immunological risks and recommends risk-based development programs.
The first clinical trial of an AAV gene therapy for type 1 diabetes is set to begin this year. The 52-week phase 1 PROGRESS study will test one-time intramuscular KRIYA-839 in adults with A1c above 7% using automated insulin delivery systems.
Tozaro, a Bedfordshire-based biotech company, has raised £6 million in funding led by Mercia Ventures to advance its Smart Polymer technology aimed at reducing the high production costs of cell and gene therapies.
Charles River Laboratories is scheduled to report Q4 2025 results on Feb. 18, with analysts raising price targets while the company navigates biotech funding pressures and announces new gene therapy collaborations.
