Could a stem cell shot before birth fix fanconi anemia?

NCT ID NCT07408583

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-phase trial tests whether giving healthy stem cells from the mother to a fetus diagnosed with Fanconi anemia can safely improve the baby's blood cell production. The treatment is a one-time injection into the fetus during pregnancy. The study will enroll 12 pregnant women and focus on safety for both mother and baby, while also checking if the stem cells help the child's bone marrow work better.

What this could mean

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

Active substance

maternal bone marrow-derived CD34+ hematopoietic stem cells

What this could lead to

If it works, this could offer a way to treat Fanconi anemia before birth, potentially reducing the need for lifelong blood transfusions or later bone marrow transplants.

What could go wrong

This is a very early, small trial (12 participants) testing safety first. The procedure carries risks for both mother and fetus, and it is unknown if the stem cells will take hold or provide lasting benefit.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

bone marrow disorder Bone Marrow Failure Disorders bone marrow failure syndrome Congenital Bone Marrow Failure Syndromes Congenital, Hereditary, and Neonatal Diseases and Abnormalities Diamond-Blackfan anemia DNA repair disease Fanconi anemia hereditary disease hereditary neoplastic syndrome inherited aplastic anemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Stanford University

    Stanford, California, 94305, United States

  • University of California, San Francisco

    San Francisco, California, 94143, United States