Research programme: autologous gene therapy - Orchard Therapeutics

Drug Profile

Research programme: autologous gene therapy - Orchard Therapeutics

Alternative Names: Ex-vivo lentiviral gene therapy - Orchard Therapeutics; OTL 201; OTL 202

Latest Information Update: 28 May 2018

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At a glance

  • Originator Central Manchester University Hospitals NHS Foundation Trust; University of Manchester
  • Developer Orchard Therapeutics
  • Class Gene therapies
  • Mechanism of Action Gene transference; 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.

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Metabolic disorders; Mucopolysaccharidosis III
  • No development reported Immunological disorders

Most Recent Events

  • 28 May 2018 No recent reports of development identified for preclinical development in Immunological-disorders in United Kingdom (Parenteral)
  • 27 Nov 2017 Orchard acquires license to develop lentivirus-based autologous ex-vivo gene therapy for Sanfilippo syndrome type B from Manchester University
  • 27 Nov 2017 Pharmacodynamics data from a preclinical trial Mucopolysaccharidosis III typeB released by Orchard
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