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A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD)

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Trial Profile

A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD)

Status: Active, no longer recruiting
Phase of Trial: Phase II

Latest Information Update: 05 Sep 2024

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At a glance

  • Drugs Atidarsagene autotemcel (Primary)
  • Indications Metachromatic leukodystrophy
  • Focus Registrational; Therapeutic Use
  • Sponsors GlaxoSmithKline; Orchard Therapeutics

    • Most Recent Events

    • 28 Aug 2024 According to an Orchard Therapeutics media release, oral presentation of this study presented at its Late stage neurometabolic Portfolio at SSIEM 2024.
    • 20 Mar 2024 According to an Orchard Therapeutics media release, company announced the details of U.S. commercial launch of Lenmeldy (atidarsagene autotemcel) for the treatment of children early-onset metachromatic leukodystrophy (MLD). The FDA approval of Lenmeldy is based on data from 37 pediatric patients with early-onset MLD, enrolled in two single-arm, open-label clinical studies or treated under European expanded access frameworks.
    • 18 Mar 2024 According to an Orchard Therapeutics media release, the FDA approval of Lenmeldy is based on data from 37 pediatric patients with early-onset MLD, enrolled in two single-arm, open-label clinical studies or treated under European expanded access frameworks, who received a one-time administration of the gene therapy and compared with natural history data.

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