MacKenzie team files IND for fetal gene therapy trial
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- Tippi MacKenzie’s team filed an investigational new drug (IND) application with the FDA to enroll five fetal patients in a first‑in‑human trial for a rare lysosomal storage disorder.
- FDA granted a waiver allowing the trial to proceed without additional animal studies, citing extensive safety data on the gene‑therapy vector from other pediatric and adult programs.
- Pre‑clinical work showed that in‑utero gene replacement cured mouse models of hemophilia and tyrosinemia, establishing proof‑of‑concept for fetal therapy.
- The proposed trial would be the earliest human application of in‑utero gene therapy, positioning the team at the forefront of prenatal genetic disease treatment.
Why it matters: The trial targets five unborn patients with a rare lysosomal storage disorder, and the FDA’s waiver eliminates months of animal testing, speeding the path to market for fetal gene‑therapy platforms.
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