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-101Latest Information Update: 02 Feb 2024
Price :
$50
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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
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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
- 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)