After decades of research, in utero gene therapy approaches first clinical trial

SAN FRANCISCO — When Tippi McKenzie was a postdoctoral researcher in the early 2000s, she and her lab colleagues conducted experiments using then-new gene replacement therapy techniques to treat genetic diseases before mice were born. It worked for me over and over again. They cured mice with hemophilia and mice with tyrosinemia. And people kept telling her that gene therapy for human fetuses was just around the corner, and would happen in just five years.

Twenty-five years later, such a reality has yet to materialize. But after promising talks with the Food and Drug Administration, McKenzie is now closer than ever.

Her team has submitted an Investigational New Drug Application (IND) to regulators seeking approval for a small trial aimed at treating five fetal patients with a rare lysosomal storage disease. The agency said the safety profile of the vectors they plan to use have already been well-characterized by other academics and companies developing gene therapies for children and adults, allowing them to avoid animal testing.

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