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OTL 300

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Drug Profile

OTL 300

Alternative Names: Autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene; GSK-2696277; OTL-300

Latest Information Update: 02 Feb 2024

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At a glance

  • Originator Fondazione Telethon; GlaxoSmithKline; Ospedale San Raffaele
  • Developer Fondazione Telethon; GSK; Ospedale San Raffaele
  • Class Gene therapies; Haematopoietic stem cells therapies; Stem cell therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

    No
  • New Molecular Entity No

Highest Development Phases

  • Phase II Beta-thalassaemia

Most Recent Events

  • 24 Jan 2024 Orchard Therapeutics has been acquired by Kyowa Kirin
  • 25 Feb 2022 OTL 300 is still in phase I/II trials for Beta-thalassaemia in Italy (Orchard Therapeutics pipeline, February 2022)
  • 15 Jul 2020 Orchard Therapeutics in-licenses lentiviral stable cell line technology from GlaxoSmithKline

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