Clinical Trial Update: Efficacy Safety Study of Gene Therapy for Sickle Cell DiseaseSCD Using Autologous CD34+ Cells Transduced ex Vivo, Carrying a Corrected Globin Gene and a Silencing RNA. [Phase 1, Phase 2]

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Summary

A Phase 1/2 clinical trial is evaluating an autologous CD34+ stem cell gene therapy with a corrected globin gene and silencing RNA for sickle cell disease; the …

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Diseases / indications

• Sickle Cell Disease

Drugs mentioned

• Autologous CD34+ gene-modified cell therapy for sickle cell disease

Snippet

Sponsor: Assistance Publique - Hôpitaux de Paris | Conditions: Sickle Cell Disease | Phase 1, Phase 2 | Status: RECRUITING