RISTOGLOGENE AUTOGETEMCEL
UNII CEL7E4NG36
Substance Identification & Data
This profile provides standardized clinical and technical data for Ristoglogene Autogetemcel, uniquely identified by the FDA Unique Ingredient Identifier (UNII) CEL7E4NG36.
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.
CEL7E4NG36
N/A
N/A
Detailed Substance Profile
Preferred Name
RISTOGLOGENE 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
13397
Sequential identifier assigned via the WHO International Nonproprietary Name program.
USAN ID
OP-137
Identifier assigned by the United States Adopted Names Council.
Substance Type
Ristoglogene 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 Ristoglogene 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
Ristoglogene Autogetemcel
Common Names & Synonyms
Autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) obtained from peripheral blood by leukapheresis, genetically modified ex vivo by electroporation of a guide RNA (gRNA) that targets the two γ-globin gene (HBG1 and HBG2) promotors and a messenger RNA (mRNA) that encodes the adenine base editor 8.8 (ABE8.8). When introduced into CD34+ cells by electroporation the gRNA binds to the translated ABE8.8 protein, which targets the promoter regions of HBG1 and HBG2, resulting in adenine to inosine modifications which subsequently leads to an A to G change. The ABE8.8 mRNA is composed of three parts: (i) an impaired form of the Streptococcus pyogenes CRISPR-associated nuclease Cas9, referred to as Cas9 D10A nickase (nCas9- D10A), (ii) an Escherichia coli derived TadA deaminase that catalyses the adenosine to inosine conversion within a small activity window along the exposed, single-stranded DNA (ssDNA) formed within the R-loop, (iii) a C terminal nuclear localization signal (NLS). The cell suspension is enriched for CD34+ cells using magnetic bead separation. Following electroporation, the cells are cultured in media containing thrombopoietin, Fms-related tyrosine kinase 3 ligand (Flt3L), and stem cell factor (SCF). The substance consists of cells with a high percentage of CD45+ and CD34+ purity and on-target editing.
Autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that uses a new base editing technology to introduce a point mutation into the HBG1 and HBG2 (HGB1/2) gene promoters to enable the re-expression of γ-globin and production of fetal hemoglobin (HbF)
RISTOGLOGENE AUTOGETEMCEL [USAN]
autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) obtained from peripheral blood by leukapheresis, genetically modified ex vivo by electroporation of a guide RNA (gRNA) that targets the two γ-globin gene (HBG1 and HBG2) promotors and a messenger RNA (mRNA) that encodes the adenine base editor 8.8 (ABE8.8). When introduced into CD34+ cells by electroporation the gRNA binds to the translated ABE8.8 protein, which targets the promoter regions of HBG1 and HBG2, resulting in adenine to inosine modifications which subsequently leads to an A to G change. The ABE8.8 mRNA is composed of three parts: (i) an impaired form of the Streptococcus pyogenes CRISPR-associated nuclease Cas9, referred to as Cas9 D10A nickase (nCas9-D10A), (ii) an Escherichia coli derived TadA deaminase that catalyses the adenosine to inosine conversion within a small activity window along the exposed, single-stranded DNA (ssDNA) formed within the R-loop, (iii) a C-terminal nuclear localization signal (NLS). The cell suspension is enriched for CD34+ cells using magnetic bead separation. Following electroporation, the cells are cultured in media containing thrombopoietin, Fms-related tyrosine kinase 3 ligand (Flt3L), and stem cell factor (SCF). The substance consists of cells with ≥85% CD45+ and ≥75% CD34+ purity and ≥75% on-target editing.
ristoglogene autogetemcel [INN]
Technical Codes
BEAM-101
