Sickle cell gene therapy trial halted after just 4 patients

NCT ID NCT04443907

Terminated Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase trial tested a gene-edited stem cell product called OTQ923 in 4 people with severe sickle cell disease. The goal was to boost fetal hemoglobin to reduce painful crises and other complications. The study was terminated early, so we have limited data on safety and effectiveness.

What this could mean

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

Active substance: OTQ923 (genome-edited stem cells)
What this could lead to: If successful, this approach could point toward a one-time treatment that reduces painful crises and other severe complications of sickle cell disease.
What could go wrong: This was a very early, small trial (only 4 participants) that was terminated, so results are limited. Gene-editing therapies also carry risks like failed engraftment or unintended genetic changes.

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

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for SICKLE CELL DISEASE are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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

Memorial Sloan Kettering Cancer Ctr

New York, New York, 10065, United States
St Jude Children's Research Hospital

Memphis, Tennessee, 38105-3678, United States
University of Chicago

Chicago, Illinois, 60637, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.