VOXERALGAGENE AUTOTEMCEL

UNII:	RZ8DCR7A94
Type:	INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:	VOXERALGAGENE AUTOTEMCEL
GSRS Full Record:	RZ8DCR7A94 i

AVR-RD-01
Autologous CD34+ hematopoietic stem cells (HSCs) transduced ex vivo with a self-inactivating lentiviral vector encoding human alpha-galactosidase A (GLA). Autologous CD34+ hematopoietic stem cells (HSCs) obtained by apheresis, transduced with a non-replicating, self-inactivating lentiviral vector encoding codon-optimized human alphagalactosidase A (GLA) under the control of the human elongation factor 1 alpha short (EFS-1 alpha) promoter and a modified woodchuck hepatitis virus post-transcriptional regulatory element (WPRE). The vector genome also contains a packaging signal, a partial gag sequence, a Rev response element (RRE), a central polypurine tract (cPPT) and a Kozak sequence. cell-based gene therapy
VOXERALGAGENE AUTOTEMCEL [USAN]
voxeralgagene autotemcel
voxeralgagene autotemcel [INN]

Note

UNIIs are generated based on scientific identity characteristics using ISO 11238 data elements. UNII availability does not imply any regulatory review or approval. Synonyms and mappings are based on the best public information available at the time of publication. Please report any problems/errors associated with this data to FDA-SRS@fda.hhs.gov.

VOXERALGAGENE AUTOTEMCEL

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