Clinical Trial Update: Safety and Efficacy of Gene Therapy of FHL Type 3 Caused by Mutations in the Human UNC13D Gene by Transplantation of a Single Dose of Autologous CD34+ Cells Transduced Ex Vivo with the UNC13D LV Vector Expressing the UNC13D CDNA [Phase 1, Phase 2]

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Summary

A Phase 1/2 clinical trial is evaluating the safety and efficacy of autologous CD34+ cells transduced with a UNC13D lentiviral vector to treat Familial Hemophag…

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Diseases / indications

• Familial Hemophagocytic Lymphohistiocytosis Type

Drugs mentioned

• UNC13D LV vector expressing UNC13D cDNA

Snippet

Sponsor: Assistance Publique - Hôpitaux de Paris | Conditions: Familial Hemophagocytic Lymphohistiocytosis Type 3 (FHL 3) | Phase 1, Phase 2 | Status: NOT_YET_RECRUITING