Experimental gene therapy aims to stop bone marrow failure in kids with rare disease
NCT ID NCT04248439
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This phase 2 trial tests a gene therapy called RP-L102 for children with Fanconi anemia subtype A, a rare genetic disorder that leads to bone marrow failure. Doctors take the child's own blood stem cells, fix the faulty gene in a lab, and infuse the corrected cells back. The goal is to restore healthy blood cell production and prevent the need for a bone marrow transplant. Only 5 children are enrolled in this early study.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
RP-L102 (gene-corrected stem cells)
What this could lead to
If successful, this could provide a one-time treatment to prevent bone marrow failure in children with Fanconi anemia subtype A, reducing the need for bone marrow transplants.
What could go wrong
This is a very small, early-phase trial with only 5 participants. It may not work for everyone, and long-term effects are unknown. The procedure also carries risks from stem cell collection and infusion.
Disclaimer
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Stanford University
Stanford, California, 94304, United States
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University of Minnesota
Minneapolis, Minnesota, 55454, United States