Gene-Editing breakthrough: could a single injection fix sickle cell?

NCT ID NCT07000318

First seen May 15, 2026 · Last updated May 15, 2026

Summary

This early-stage study tests a new gene therapy called CS-206 for people with severe sickle cell disease. The treatment uses a patient's own blood stem cells, which are modified with a gene-editing tool to correct the disease. Five participants aged 12 to 18 will receive a single dose, and researchers will monitor safety and whether it reduces painful crises.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Children's Hospital of Fudan University

    Shanghai, Shanghai Municipality, 201102, China

Conditions

Explore the condition pages connected to this study.