ADA SCID gene therapy - Orchard Therapeutics

Drug Profile

ADA SCID gene therapy - Orchard Therapeutics

Alternative Names: Autologous cryopreserved EFS-ADA LV CD34+ cells - Orchard Therapeutics; Autologous ex-vivo gene therapy - Orchard Therapeutics; EF1αS-ADA lentiviral gene therapy - Orchard Therapeutics; EFS-ADA LV; Lentiviral gene therapy - Orchard Therapeutics; OTL-101

Latest Information Update: 07 Jun 2017

Price : $50

At a glance

  • Originator Orchard Therapeutics
  • Developer Great Ormond Street Hospital; Orchard Therapeutics; University of California at Los Angeles
  • Class Gene therapies; Stem cell therapies
  • Mechanism of Action Adenosine deaminase stimulants; Gene transference
  • Orphan Drug Status

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

    Yes - Adenosine deaminase deficiency
  • New Molecular Entity No

Highest Development Phases

  • Phase II/III Adenosine deaminase deficiency

Most Recent Events

  • 30 May 2017 Orchard Therapeutics signs a manufacturing services agreement with PCT, a Caladrius company for ADA SCID gene therapy in treatment of Adenosine deaminase deficiency
  • 01 Dec 2016 University of California, Los Angeles, University College, London and Orchard therapeutics initiate a phase I/II trial for Adenosine deaminase deficiency in USA (IV) (NCT02999984)
  • 29 Nov 2016 Oxford BioMedica will develop and supply lentiviral vectors to Orchard Therapeutics for Adenosine deaminase deficiency
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