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.
University of Missouri researchers released PSBench, a database of 1.4 million annotated protein structure models verified by independent experts. The resource aims to improve AI assessment of protein models as biologic drug discovery moves toward data-driven design.
Johnson & Johnson invests over $1 billion in a new Pennsylvania cell therapy manufacturing facility for cancer and neurological diseases. The expansion addresses growing demand for advanced therapies while highlighting workforce training challenges in GMP environments. Other developments include new cell line technology for viral vector production and CRISPR-edited CAR-T manufacturing partnerships.
The global mRNA and next-generation vaccine platforms market is expected to grow from $39.69 billion in 2025 to $82.61 billion by 2030 at a 15.8% CAGR, driven by personalized medicine demand and infectious disease applications, while safety concerns and regulatory scrutiny continue.
UCLA scientists developed a lipid nanoparticle-based gene editing system that successfully inserts a full-length healthy CFTR gene into human airway cells, offering potential treatment for cystic fibrosis patients unresponsive to current therapies.
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.
