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Atidarsagene autotemcel - Orchard Therapeutics

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Drug Profile

Atidarsagene autotemcel - Orchard Therapeutics

Alternative Names: ARSA gene therapy - GSK/Fondazione Telethon/Ospedale San Raffaele; ARSA transduced autologous CD34+ stem cells - GSK/Fondazione Telethon/Ospedale San Raffaele; Arylsulfatase A gene therapy -GSK/Fondazione Telethon/Ospedale San Raffaele; Ex vivo stem cell gene therapy - GSK/Fondazione Telethon/Ospedale San Raffaele; GSK 2696274; Lenmeldy; Libmeldy; OTL-200

Latest Information Update: 07 Nov 2025

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At a glance

  • Originator Fondazione Telethon; GlaxoSmithKline; Ospedale San Raffaele
  • Developer Orchard Therapeutics; The San Raffaele Telethon Institute for Gene Therapy (JV)
  • Class Gene therapies; Haematopoietic stem cells therapies
  • Mechanism of Action Arylsulfatase replacements; Cell replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Metachromatic leukodystrophy
  • New Molecular Entity No

Highest Development Phases

  • Marketed Metachromatic leukodystrophy

Most Recent Events

  • 28 Oct 2025 Atidarsagene autotemcel receives Orphan Regenerative Medicine Product Designation for Metachromatic leukodystrophy in Japan
  • 28 Oct 2025 Kyowa Kirin plans clinical trial for Metachromatic leukodystrophy (In children) in Japan
  • 12 Feb 2025 Launched for Metachromatic leukodystrophy (In children) in USA (IV) prior to February 2025

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