Gene-Editing breakthrough? new therapy aims to stop sickle cell pain crises

NCT ID NCT06565026

Recruiting now Disease control Sponsor: CorrectSequence Therapeutics Co., Ltd Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-phase trial is testing a new treatment called CS-101 for severe sickle cell disease. It uses a person's own blood stem cells, which are gene-edited in a lab to correct the disease, then given back as a one-time infusion. The study will enroll 5 people aged 12 to 35 and will primarily check for safety and whether the treatment reduces severe pain episodes.

What this could mean

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

Active substance: CS-101 (gene-edited stem cells)
What this could lead to: If successful, this could offer a one-time treatment that reduces painful crises and hospitalizations for people with severe sickle cell disease.
What could go wrong: This is a very early, small trial (5 people) focused on safety. The therapy may not work, and there are risks from the stem cell transplant procedure itself.

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

The First Affiliated Hospital of Guangxi Medical University
RECRUITING

Nanning, Guangxi, China

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