Elivaldogene Autotemcel

Preferred Label : Elivaldogene Autotemcel;

NCIt synonyms : Eli-cel; Lenti-D (TM);

NCIt definition : A gene replacement therapy agent composed of autologous cluster of differentiation 34 (CD34)-positive human hematopoietic stem cells (HSCs) transduced ex vivo with Lenti-D lentiviral vector (LVV), a replication-incompetent, self-inactivating LVV carrying ABCD1 complementary DNA (cDNA) that encodes normal adrenoleukodystrophy protein (ALD protein; ALDP), that can be used to treat early cerebral adrenoleukodystrophy (CALD). Upon administration, elivaldogene autotemcel engrafts in the bone marrow and differentiates into various cell types, including monocytes, that produce functional copies of the ABCD1 cDNA and thus functional ALDP. Functional ALDP degrades very long chain fatty acids (VLCFAs), which prevents accumulation of VLCFAs and may decrease or prevent further inflammation, demyelination and nerve damage. This slows the progression of neurologic dysfunction in patients with early, active cerebral adrenoleukodystrophy (CALD).;

UNII : KUM75TD6SG;

Drug name : Skysona;

NCI Metathesaurus CUI : CL1406003;

Details

Origin ID

C171808;

UMLS CUI

C5419314;

Semantic type(s)
- Cell [UMLS semantic type]
UMLS correspondences (same concept)
- elivaldogene autotemcel [ATC Code]
Validated automatic mappings to BTNT
- elivaldogene autotemcel [ATC Code]
concept_is_in_subset
- FDA Established Names and Unique Ingredient Identifier Codes Terminology [NCIt concept]

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