Drug Profile
Elivaldogene autotemcel - bluebird bio
Alternative Names: Adrenoleucodystrophy gene therapy - bluebird bio; eli-cel; Lenti-D; Lenti-D ABCD1 gene therapy - bluebird bio; Lenti-D ACALD gene therapy - bluebird bio; Lenti-D adrenoleukodystrophy gene therapy - bluebird bio; Lenti-D ALD gene therapy - bluebird bio; Lenti-D ALDP gene therapy - bluebird bio; Lenti-D AMN gene therapy - bluebird bio; Lenti-D ATP binding cassette sub family-D (ALD) member 1 gene therapy - bluebird bio; Lenti-D CCALD gene therapy - bluebird bio; Lenti-D X-ALD gene therapy - bluebird bio; SKYSONALatest Information Update: 09 Nov 2023
Price :
$50
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At a glance
- Originator Genetix Pharmaceuticals
- Developer bluebird bio
- Class Adrenoleucodystrophy gene therapies; Gene therapies; Haematopoietic stem cells therapies
- Mechanism of Action ALDP expression stimulants; Cell replacements
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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
- Marketed Adrenoleucodystrophy
Most Recent Events
- 07 Nov 2023 Launched for Adrenoleucodystrophy (In adolescents, In children) in USA (IV)
- 07 Nov 2023 Launched for Adrenoleucodystrophy (In adolescents, In children, In infants, In neonates) in European Union (IV)
- 07 Nov 2023 Launched for Adrenoleucodystrophy (In adolescents, In children, In infants, In neonates) in Iceland (IV)