Fetal Gene Therapy Makes Strides Toward the Clinic

Scientists have had prenatal gene therapy in their sights for decades; by treating the fetus in utero, they can potentially prevent the long-term damage caused by severe, early-onset genetic disorders. Despite its success in preclinical studies, the method has not been successfully translated into human patients yet. However, that may soon change.

Last week, fetal surgeon Tippi MacKenzie of the University of California, San Francisco described the design of a phase I, first-in-human trial of a gene therapy for the treatment of GM1 gangliosidosis, a rare, inherited lysosomal storage disorder, in prenatal patients. “Evidence of safety and perhaps efficacy with this disease could allow us to unlock the field of clinical fetal gene therapy for related conditions,” MacKenzie remarked in her presentation during the ‘In Utero Therapeutics: Engineering Fetal Futures with Cell and Gene Therapy’ session at the 2026 annual meeting of the American Society of Gene and Cell Therapy.

Gaps in Clinical Translation of Fetal Gene Therapy

Why has the clinical translation of fetal gene therapy been unsuccessful to date? According to MacKenzie, to move any fetal molecular...