Clinical Trial Update: Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene [Phase 1, Phase 2]

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Summary

Phase 1/2 clinical trial assessing autologous CD34+ hematopoietic stem cells transduced with an ADA gene lentiviral vector in ADA-SCID; study completed with res…

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Diseases / indications

• Ada-scid

Drugs mentioned

• Autologous CD34+ hematopoietic stem cells transduced ex vivo
• Elongation Factor 1 Alpha Shortened (EFS) lentiviral vector encoding the human ADA gene (ADA gene lentiviral vector)

Snippet

Sponsor: University of California, Los Angeles | Conditions: ADA-SCID | Phase 1, Phase 2 | Status: COMPLETED | Results posted: 2021-09-20