ATAGLOGENE AUTOGETEMCEL
UNII X9AT6SDG44
Substance Identification & Data
This profile provides standardized clinical and technical data for Ataglogene Autogetemcel, uniquely identified by the FDA Unique Ingredient Identifier (UNII) X9AT6SDG44.
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.
X9AT6SDG44
N/A
N/A
Detailed Substance Profile
Preferred Name
ATAGLOGENE AUTOGETEMCEL
Official standardized name for this substance within the FDA UNII nomenclature system.
NCI Thesaurus
National Cancer Institute reference terminology for clinical and research data.
INN ID
13584
Sequential identifier assigned via the WHO International Nonproprietary Name program.
Substance Type
Ataglogene Autogetemcel
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 Ataglogene Autogetemcel. 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
Ataglogene Autogetemcel
Common Names & Synonyms
ataglogene autogetemcel [INN]
autologous CD34+ hematopoietic stem cells obtained by apheresis, electroporated with a multi-component CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9)-guide RNA (gRNA) transformer base editor nucleoprotein complex that targets the promoter regions of the two γ-globin genes (HBG1 and HBG2) to disrupt binding of the transcriptional repressor BCL11A (B-cell lymphoma/leukemia 11A), leading to the re-expression of γ-globin and increased production of foetal haemoglobin (HbF). The transformer base editing system comprises two mRNAs and two single guide RNAs. The first mRNA (mRNA-L) encodes an impaired form of the Streptococcus pyogenes clustered regularly interspaced short palindromic repeats (CRISPR) associated protein 9 and is referred to as Cas9 nickase D10A. The second mRNA (mRNA-EK) encodes three independent proteins, separated by 2A peptides; the first protein (MCP-UGI-mA3CDA1-mA3dCDI), is a fusion protein comprising (5' to 3') an RNA aptamer binding protein [bacteriophage MS2 coat protein (MCP)], a uracil DNA glycosylase inhibitor (UGI), the mouse apolipoprotein B mRNA-editing complex 3 (APOBEC3, mA3) cytidine deaminase domain 1 (mA3CDA1), a tobacco etch virus (TEV) protease cleavage site, and the mouse APOBEC3 deoxycytidine deaminase inhibitor domain (dCDI) (mA3dCDI). The second protein (N22p-TEVc) contains an RNA aptamer binding protein (bacteriophage N peptide 1-22) fused with the C-terminal fragment of the tobacco etch virus protease (TEVc). The third protein (TEVn) comprises the N-terminal fragment of the TEV protease. The first single guide RNA is a helper gRNA (hsgRNA) that guides nCas9 to the first target site and contains an MS2 hairpin to recruit the MCP-UGI-mA3CDA1-mA3dCDI fusion protein. The second sgRNA is a modifying gRNA (msgRNA) that guides a second nCas9 to the gene editing target site and contains box B hairpins to recruit the N22p-TEVc protein. When the two guide RNAs bind on-target, the two halves of the TEV protease (TEVn and TEVc) reconstitute, and the protease can cleave off the deoxycytidine deaminase inhibitor (mA3dCDI) from the mRNA editing cytidine deaminase domaine 1 (mA3CDA1) to edit the transcriptional repressor BCL11A binding domain of the γ-globin promoter regions. The apheresis material is enriched in CD34+ cells using immunomagnetic microbeads, gene-edited and then cultured in the presence of thrombopoietin (TPO), stem cell factor (SCF) and Fms-like tyrosine kinase 3 ligand (Flt-3L). The final substance contains ≥90% CD34+ cells and has a target gene editing efficiency of ≥30%
