SAN FRANCISCO — In the early 2000s, Tippi MacKenzie, then a postdoctoral fellow, and her colleagues explored the potential of gene replacement therapy to address inherited disorders in mice before birth. Their efforts repeatedly succeeded, curing mice afflicted with hemophilia and tyrosinemia. Throughout this time, MacKenzie was frequently told that gene therapy in human fetuses was imminent, only five years away.
Now, 25 years later, this vision still has not become reality. However, after encouraging discussions with the Food and Drug Administration (FDA), MacKenzie is closer to achieving this goal than ever before.
Her team has filed an investigational new drug (IND) application with the FDA, seeking approval for a small trial aimed at treating five fetal patients with a rare lysosomal storage disorder. The FDA indicated that they could bypass animal testing due to the well-established safety profile of the vector they plan to use, which has been characterized by other researchers and companies developing gene therapies for children and adults.
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