Gene therapy aims to cure Transfusion-Dependent thalassemia in a single shot

NCT ID NCT07721480

Not yet recruiting ⭐️ CURE ⭐️ Sponsor: Prince of Wales Hospital, Shatin, Hong Kong Source: ClinicalTrials.gov ↗

First seen Jul 23, 2026 · Last updated Jul 24, 2026 · Updated 1 time

Summary

This phase 2 trial tests a one-time gene therapy called HGI-001 in people with transfusion-dependent β-thalassemia, a severe blood disorder that requires regular blood transfusions. The therapy takes the patient's own blood stem cells, modifies them outside the body to produce healthy hemoglobin, and infuses them back. The goal is to see if this approach can allow participants to go without transfusions for at least a year, potentially offering a lasting cure.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: a one-time gene therapy that modifies the patient's own blood stem cells to produce healthy hemoglobin
What this could lead to: If successful, this could offer a one-time cure for transfusion-dependent β-thalassemia, eliminating the need for lifelong blood transfusions and reducing complications from iron overload.
What could go wrong: This is an early-phase trial with only 6 participants, so results may not apply to everyone. Gene therapies carry risks such as immune reactions or long-term side effects that are still being monitored.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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