Gene therapy shows promise for sickle cell disease in small trial

NCT ID NCT02247843

Completed Disease control Sponsor: Donald B. Kohn, M.D. Source: ClinicalTrials.gov ↗

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

Summary

This early-phase clinical trial tested a gene therapy for adults with severe sickle cell disease. The treatment involved taking the patient's own blood stem cells, modifying them with a harmless virus to carry a healthy gene, and infusing them back after mild chemotherapy. Only 4 people took part, and the main goal was to check safety. Early results suggest the approach may reduce sickle cell complications, but larger studies are needed to confirm.

What this could mean

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

Active substance: gene-modified stem cells (Lenti/βAS3-FB)
What this could lead to: If successful, this could lead to a one-time treatment that reduces or eliminates sickle cell disease symptoms without needing a donor.
What could go wrong: This was a very small early-phase trial with only 4 participants. Long-term safety and effectiveness are not yet proven, and there are risks from the chemotherapy used before the transplant.

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Conditions

The condition(s) this trial relates to.

sickle cell disease

As listed by the trial registrant

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

SICKLE CELL DISEASE

Contacts and locations

Locations

University of California, Los Angeles (UCLA)

Los Angeles, California, 90095, United States

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