ATIDARSAGENE AUTOTEMCEL
UNII EPP8G99QG4

Substance Identification & Data

This profile provides standardized clinical and technical data for Atidarsagene Autotemcel, uniquely identified by the FDA Unique Ingredient Identifier (UNII) EPP8G99QG4.

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

EPP8G99QG4

CAS Registry Number

N/A

RxNorm Concept ID

N/A

Detailed Substance Profile

Preferred Name

ATIDARSAGENE AUTOTEMCEL

Official standardized name for this substance within the FDA UNII nomenclature system.

Molecular Formula

EPP8G99QG4

The specific atomic composition represented as a molecular structure string.

NCI Thesaurus

C180638

National Cancer Institute reference terminology for clinical and research data.

INN ID

11282

Sequential identifier assigned via the WHO International Nonproprietary Name program.

USAN ID

JK-136

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 Atidarsagene 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

ATIDARSAGENE AUTOTEMCEL

Common Names & Synonyms

ATIDARSAGENE AUTOTEMCEL [INN]

ATIDARSAGENE AUTOTEMCEL [USAN]

ATIDARSAGENE AUTOTEMCEL [WHO-DD]

AUTOLOGOUS CD34+ CELLS, CONTAINING HEMATOPOIETIC STEM CELLS (HSCS), TRANSDUCED WITH A LVV ENCODING THE HUMAN ARYLSULFATASE A (ARSA) GENE

AUTOLOGOUS CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS TRANSDUCED EX VIVO WITH A REPLICATION-INCOMPETENT LENTIVIRAL VECTOR ENCODING HUMAN ARYLSULFATASE A (ARSA). AUTOLOGOUS CD34+ HEMATOPOIETIC STEM AND PROGENITOR CELLS (HSPC), OBTAINED FROM BONE MARROW (BM) OR MOBILIZED PERIPHERAL BLOOD (MPB), TRANSDUCED EX VIVO WITH A REPLICATION-INCOMPETENT PSEUDOTYPED HIV-I BASED LENTIVIRAL VECTOR ENCODING THE HUMAN ARYLSULFATASE A (ARSA) TRANSGENE UNDER THE CONTROL OF A HUMAN PHOSPHOGLYCERATE KINASE (PGK) PROMOTER. THE VECTOR IS PSEUDOTYPED WITH THE VESICULAR STOMATITIS VIMS (VSV) ENVELOPE GLYCOPROTEIN. THE VECTOR GENOME ALSO CONTAINS A MUTATED WOODCHUCK HEPATITIS VIMS POST-TRANSCRIPTIONAL REGULATORY ELEMENT (WPRE), A FRAGMENT OF THE HIV-I GAG, THE HIV-I REV RESPONSE ELEMENT (RRE), THE HIV-1 CENTRAL POLYPURINE TRACT (CPPT) AND A FRAGMENT OF NEF. THE CD34+ CELL POPULATION CORRESPONDS TO AN HETEROGENOUS POPULATION OF STEM AND PROGENITOR CELLS, SOME EXPRESSING THE CD90 AND/OR CD133 MARKERS.