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A Proof of Concept Clinical Trial to Investigate OTL-300 in Transfusion-Dependent Beta-Thalassemia Patients

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Trial Profile

A Proof of Concept Clinical Trial to Investigate OTL-300 in Transfusion-Dependent Beta-Thalassemia Patients

Status: Recruiting
Phase of Trial: Phase I

Latest Information Update: 04 Jul 2023

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At a glance

  • Drugs OTL 300 (Primary) ; Thiotepa; Treosulfan
  • Indications Beta-thalassaemia
  • Focus Adverse reactions; Proof of concept; Therapeutic Use

    • Most Recent Events

    • 29 Apr 2019 According to the Orchard Therapeutics media release, data from this trial will be featured in an oral presentation at the 22nd American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Washington, D.C. The presentation will report the updated safety and efficacy results of OTL-300 in nine TDT patients.
    • 29 Apr 2019 Results published in the Orchard Therapeutics media release.
    • 15 Apr 2019 According to the Orchard Therapeutics media release, data from this trial will be published online in an abstract via the American Society of Gene & Cell Therapy website and full data set will be featured in an oral presentation at the 22nd ASGCT Annual Meeting 2019.

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