Elivaldogene Autotemcel

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).

Also known as Eli-cel, ELIVALDOGENE AUTOTEMCEL, Lenti-D (TM), Skysona — per NCIT

Also identified as

• ATC A16AX21 per RXNORM
• NCIT NCIT:C171808 per NCIT
• UNII KUM75TD6SG per RXNORM

Indications

Organ Effects

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