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

Latest Information Update: 07 Dec 2016

Price : $50

At a glance

  • Originator Orchard Therapeutics
  • Class Gene 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

  • Preclinical Immunological disorders; Metabolic disorders; Mucopolysaccharidosis III

Most Recent Events

  • 29 Nov 2016 Mucopolysaccharidosis-IIIA (MPS-IIIA or Sanfilippo Syndrome type A) and undisclosed follow-on indications
  • 21 Oct 2016 Orchard Therapeutics plans a first-in-man trial for Mucopolysaccharidosis III (Orchard Therapeutics website, October 2016)
  • 19 Oct 2016 Preclinical trials in Metabolic disorders in United Kingdom (Parenteral)
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