The field of gene therapies requires extensive investment, transparency and sound regulatory frameworks. These measures are intended to help overcome recent challenges in this field. The goal is to continue showing new technologies for the benefit of patients. The article was published in Nature Medicine online on March 13, 2026 with doi: 10.1038/s41591-026-04311-y. Genetic therapies face challenges such as delayed regulatory approvals and high manufacturing costs. Innovations in automation, continuous vector production and robust clinical validation are needed. Regulatory bodies such as the EMA update the GMP Part IV guidelines for safety and reliability.