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OTL 201

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Drug Profile

OTL 201

Alternative Names: OTL-201

Latest Information Update: 02 Feb 2024

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At a glance

  • Originator Central Manchester University Hospitals NHS Foundation Trust; University of Manchester
  • Developer Orchard Therapeutics; University of Manchester
  • Class Gene therapies; Haematopoietic stem cells therapies
  • Mechanism of Action Cell replacements; N-sulfoglucosamine sulfohydrolase expression stimulants
  • Orphan Drug Status

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

    Yes - Mucopolysaccharidosis III
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Mucopolysaccharidosis III

Most Recent Events

  • 24 Jan 2024 Orchard Therapeutics has been acquired by Kyowa Kirin
  • 03 Aug 2023 Updated efficacy data from a phase I/II trial in mucopolysaccharidosis type IIIA (MPS-IIIA) released by Orchard Therapeutics
  • 12 Dec 2022 Efficacy and adverse events data from a phase-I/II trial in Mucopolysaccharidosis-III released by Orchard Therapeutics

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