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Drug Profile

OTL 103

Alternative Names: GSK-2696275; OTL-103; WAS gene therapy - Orchard Therapeutics; WAS transduced autologous CD34+ stem cells - Orchard Therapeutics; WAS transduced CD34+ stem cells

Latest Information Update: 02 Feb 2024

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At a glance

  • Originator Fondazione Telethon; GlaxoSmithKline; Ospedale San Raffaele
  • Developer Orchard Therapeutics; Ospedale San Raffaele
  • Class Gene therapies; Haematopoietic stem cells therapies
  • Mechanism of Action Cell replacements
  • Orphan Drug Status

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

    Yes - Wiskott-Aldrich syndrome
  • New Molecular Entity No

Highest Development Phases

  • Phase III Wiskott-Aldrich syndrome

Most Recent Events

  • 24 Jan 2024 Orchard Therapeutics has been acquired by Kyowa Kirin
  • 04 Oct 2023 Ospedale San Raffaele completes a phase I/II trial in Wiskott Aldrich Syndrome in Italy (NCT01515462)
  • 16 Feb 2022 Phase-III clinical trials in Wiskott-Aldrich syndrome (In adolescents, In children, In infants, In adults) in USA (IV) (NCT03837483, EudraCT2018-003842-18)

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