Gene therapy is making significant strides, particularly for rare genetic diseases. For example, an investigational gene therapy has successfully restored immune function in children with severe leukocyte adhesion deficiency-I (LAD-I) in an international trial (UCL, May 2025). Beyond rare diseases, researchers are also developing new delivery systems, such as DNA-loaded lipid nanoparticles (LNPs), which could make gene therapy applicable to common chronic diseases by improving safety and sustained protein production (Penn Medicine, March 2025). While AAV-based gene therapies face some challenges leading to pharma pullback in some areas, the overall field, including advancements in CRISPR-Cas9 gene-editing tools, continues to grow, with increased investment and new approvals.