A non-randomized trial to evaluate the safety and efficacy of ex-vivo EFS lentiviral-vector-based gene therapy.

Trial Profile

A non-randomized trial to evaluate the safety and efficacy of ex-vivo EFS lentiviral-vector-based gene therapy.

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 18 Oct 2016

At a glance

  • Drugs ADA-SCID gene therapy (Primary)
  • Indications Adenosine deaminase deficiency
  • Focus Adverse reactions; Therapeutic Use
  • Most Recent Events

    • 18 Oct 2016 New trial record
    • 22 Sep 2016 Pooled clinical data (n=32) from this and another study (Profile 277537) published in an Orchard Therapeutics media release.
    • 21 Sep 2016 According to Orchard Media release, clinical data from this study will be presented at the 17th biennial meeting of the European Society for Immunodeficiencies (ESID) 2016 and at the European Society of Gene and Cell Therapy (ESGCT) conference.
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