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Simoladagene autotemcel - Orchard Therapeutics

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Drug Profile

Simoladagene autotemcel - Orchard Therapeutics

Alternative Names: ADA gene therapy - Orchard Therapeutics; ADA SCID gene therapy -Orchard Therapeutics; Adenosine deaminase gene therapy - Orchard Therapeutics; Autologous cryopreserved EFS-ADA LV CD34 cells; Autologous ex-vivo lentiviral adenosine deaminase gene therapy - Orchard Therapeutics; OTL-101

Latest Information Update: 02 Feb 2024

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At a glance

  • Originator University College London; University of California at Los Angeles
  • 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

  • 24 Jan 2024 Orchard Therapeutics has been acquired by Kyowa Kirin
  • 10 Feb 2023 Great Ormond Street Hospital and Orchard Therapeutics completes a phase I/II trial in Adenosine deaminase deficiency (In children, In adolescents, In neonates, In infants) in United Kingdome (NCT03765632)
  • 18 Nov 2021 Orchard Therapeutics terminates a phase-II/III trial for Adenosine deaminase deficiency in USA, due to business reasons (NCT04140539)

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