Gene-Editing hope: could BIVV003 free sickle cell patients from pain crises?

NCT ID NCT03653247

Completed Disease control Sponsor: Sangamo Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This early-phase trial tested a new gene therapy called BIVV003 in 7 adults with severe sickle cell disease. The treatment uses the patient's own stem cells, which are gene-edited in a lab and then infused back after chemotherapy. The main goals were to check safety and see if the cells engraft successfully. Because it's a small, early study, results are preliminary and more research is needed.

What this could mean

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

Active substance: BIVV003 (gene-edited stem cells)
What this could lead to: If successful, this could point toward a one-time treatment that reduces or eliminates sickle cell crises without needing a donor.
What could go wrong: This is a very early, small study (only 7 people) focused on safety. The gene-editing approach is new, and long-term effects or failure to control the disease are possible.

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

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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

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States
Investigational Site Number 101

Bethesda, Maryland, 20892, United States
Karmanos Cancer Institute

Detroit, Michigan, 48201, United States
UCSF Benioff Children's Hospital

Oakland, California, 94609, United States
University of California Davis Comprehensive Cancer Center

Sacramento, California, 95817, United States

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