Gene therapy breakthrough? new treatment aims to fix sickle cell at the source

NCT ID NCT04819841

Recruiting now Disease control Sponsor: Kamau Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 02, 2026 · Updated 1 time

Summary

This early-stage trial is testing a new gene therapy called nula-cel for people with severe sickle cell disease. The therapy modifies a patient's own blood stem cells to produce normal hemoglobin instead of sickle hemoglobin. About 15 participants aged 12 to 40 will receive the treatment after a conditioning regimen. The main goals are to check safety and see if the therapy can reduce or eliminate sickle cell complications.

What this could mean

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

Active substance: nula-cel (a gene therapy that modifies a patient's own blood stem cells to produce normal hemoglobin)
What this could lead to: If it works, this could provide a one-time treatment that controls severe sickle cell disease by enabling the body to make healthy hemoglobin instead of sickle hemoglobin.
What could go wrong: This is a very early, first-in-human trial with only 15 participants. The conditioning regimen has serious risks, and the gene therapy may not work as expected or could cause side effects. Long-term safety and effectiveness are unknown.

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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 Hospital Los Angeles
RECRUITING

Los Angeles, California, 90027, United States
Columbia University Irving Medical Center
RECRUITING

New York, New York, 10032, United States
Lucile Packard Children's Hospital
RECRUITING

Palo Alto, California, 94304, United States
Memorial Sloan Kettering
RECRUITING

New York, New York, 10065, United States
Nationwide Children's Hospital
RECRUITING

Columbus, Ohio, 43205, United States
Washington University
RECRUITING

St Louis, Missouri, 63110, United States

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