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: 24 Aug 2017

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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

  • 22 Aug 2017 The UK MHRA designates Promising Innovative Medicine designation to ADA-SCID gene therapy for Adenosine deaminase deficiency in the United Kingdom
  • 24 Jul 2017 ADA SCID gene therapy - Orchard Therapeutics receives Breakthrough Therapy status for Adenosine deaminase deficiency in USA before July 2017
  • 24 Jul 2017 ADA SCID gene therapy - Orchard Therapeutics receives Rare Paediatric Disease designation for Adenosine deaminase deficiency in USA
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