EXAGAMGLOGENE AUTOTEMCEL
UNII S53L777GM8

Substance Identification & Data

This profile provides standardized clinical and technical data for Exagamglogene Autotemcel, uniquely identified by the FDA Unique Ingredient Identifier (UNII) S53L777GM8.

Technical mappings include the Chemical Abstracts Service (CAS) Registry Number N/A and the RxNorm Concept ID (RxCUI) N/A. Explore the sections below for detailed nomenclature and a complete directory of NDC-listed products containing this ingredient.

FDA UNII Code
S53L777GM8
CAS Registry Number
N/A
RxNorm Concept ID
N/A

Detailed Substance Profile

Preferred Name
EXAGAMGLOGENE AUTOTEMCEL
Official standardized name for this substance within the FDA UNII nomenclature system.
Molecular Formula
S53L777GM8
The specific atomic composition represented as a molecular structure string.
NCI Thesaurus
National Cancer Institute reference terminology for clinical and research data.
INN ID
11560
Sequential identifier assigned via the WHO International Nonproprietary Name program.
USAN ID
KL-107
Identifier assigned by the United States Adopted Names Council.
Substance Type
structurallyDiverse
ISO 11238 classification category (e.g., Chemical, Polymer, Protein).
ITIS TSN
180092
Taxonomic Serial Number for species identified in the Integrated Taxonomic Information System.
NCBI Taxonomy
9606
Unique numeric identifier used to specify biological species in the NCBI database.

Synonyms and Nomenclature

This section provides a complete list of nomenclature and identifier mappings for Exagamglogene Autotemcel. Identifiers are organized into official regulatory terms, commercial trade names, and technical systematic synonyms used to ensure accurate identification across clinical pharmaceutical databases, regulatory filings, and electronic health records.

FDA Official Name

EXAGAMGLOGENE AUTOTEMCEL

Common Names & Synonyms

AUTOLOGOUS CD34+ CELLS ISOLATED FROM MOBILISED PERIPHERAL BLOOD BY POSITIVE SELECTION, MODIFIED BY CRISPR/CAS9 (CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS/CRISPR-ASSOCIATED PROTEIN 9) MEDIATED GENE EDITING CONSISTING OF A GUIDE RNA (GRNA) INTRODUCED TRANSIENTLY AS RIBONUCLEOPROTEIN (RNP) COMPLEX, TARGETING THE ERYTHROID LINEAGE-SPECIFIC ENHANCER REGION OF BCL11A (B-CELL LYMPHOMA/LEUKEMIA 11A). THE SITE-SPECIFIC CLEAVAGE BY CAS9 FORMS A DOUBLE STRAND BREAK (DSB), WHICH IS SUBSEQUENTLY REPAIRED BY NONHOMOLOGOUS END-JOINING (NHEJ), LEADING TO THE TRANSCRIPTIONAL REPRESSION OF BCL11A, A REPRESSOR OF .GAMMA.-GLOBIN GENE TRANSCRIPTION
EXAGAMGLOGENE AUTOTEMCEL [INN]
EXAGAMGLOGENE AUTOTEMCEL [USAN]
EXAGAMGLOGENE AUTOTEMCEL [WHO-DD]

Technical Codes

CTX001
CTX-001

NDC Products Containing EXAGAMGLOGENE AUTOTEMCEL

This unique ingredient identifier is listed as an ingredient or substance in the following products in the NDC directory: