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Efficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to ADA Deficiency

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Trial Profile

Efficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to ADA Deficiency

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 08 Aug 2022

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At a glance

  • Drugs Simoladagene autotemcel (Primary) ; Busulfan; Elapegademase
  • Indications Adenosine deaminase deficiency
  • Focus Therapeutic Use
  • Acronyms UCLA Cryo Study
  • Sponsors Orchard Therapeutics

    • Most Recent Events

    • 11 May 2021 Results (n=50) from NCT01852071, NCT02999984, and NCT01380990 assessing overall and event-free survival in autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency, published in the New England Journal of Medicine
    • 11 May 2021 Data from three Phase 1/2 clinical studies (n=40), and a compassionate use program (n=10) presented in an Orchard Media Release.
    • 11 May 2021 According to an Orchard Therapeutics media release, data from three Phase 1/2 clinical studies (n=40), and a compassionate use program (n=10) published in the New England Journal of Medicine (NEJM) .
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