Gene therapy, which involves replacing defective genes to treat diseases, has been a promising but challenging field. Early optimism in the mid-1980s to early 1990s was dampened by issues such as poor gene expression and immune complications. However, recent advancements, including improved gene delivery methods and better understanding of viral vectors, show progress. The American Society of Gene Therapy (ASGT) meeting highlighted the importance of a solid biological understanding for clinical efficacy. The theme of this year's meeting was stem cells, reflecting recent advances and ethical debates. Despite regulatory hurdles and the need for further research, there is hope for successful gene therapy treatments, with one notable success in X-linked severe combined immunodeficiency. The FDA is also consolidating its efforts to regulate gene therapy, which may impact the regulatory environment.Gene therapy, which involves replacing defective genes to treat diseases, has been a promising but challenging field. Early optimism in the mid-1980s to early 1990s was dampened by issues such as poor gene expression and immune complications. However, recent advancements, including improved gene delivery methods and better understanding of viral vectors, show progress. The American Society of Gene Therapy (ASGT) meeting highlighted the importance of a solid biological understanding for clinical efficacy. The theme of this year's meeting was stem cells, reflecting recent advances and ethical debates. Despite regulatory hurdles and the need for further research, there is hope for successful gene therapy treatments, with one notable success in X-linked severe combined immunodeficiency. The FDA is also consolidating its efforts to regulate gene therapy, which may impact the regulatory environment.