RENIZGAMGLOGENE AUTOGEDTEMCEL
UNII 4A4FLA6DZA

Substance Identification & Data

This profile provides standardized clinical and technical data for Renizgamglogene Autogedtemcel, uniquely identified by the FDA Unique Ingredient Identifier (UNII) 4A4FLA6DZA.

Technical mappings include the Chemical Abstracts Service (CAS) Registry Number 2923537-23-7 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

4A4FLA6DZA

CAS Registry Number

2923537-23-7

RxNorm Concept ID

N/A

Detailed Substance Profile

Preferred Name

RENIZGAMGLOGENE AUTOGEDTEMCEL

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

NCI Thesaurus

C203030

National Cancer Institute reference terminology for clinical and research data.

INN ID

12733

Sequential identifier assigned via the WHO International Nonproprietary Name program.

USAN ID

MN-162

Identifier assigned by the United States Adopted Names Council.

Substance Type

Renizgamglogene Autogedtemcel

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 Renizgamglogene Autogedtemcel. 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

Renizgamglogene Autogedtemcel

Common Names & Synonyms

Autologous human CD34+ hematopoietic stem/precursor cells (HSPCs), obtained from peripheral blood by leukapheresis from plerixaformobilized sickle cell disease (SCD) patients or granulocyte colony stimulating factor (G-CSF) and plerixafor-mobilized transfusion dependent betathalassemia (TDT) patients, genetically modified ex vivo by CRISPR/Cas12a (clustered regularly interspaced palindromic repeats/modified Acidaminococcus sp. Cas12a) endonuclease complexed with a guide RNA (gRNA) that targets the CCAAT-box region of both gamma globin gene (HBG1 and HGB2) promoters on chromosome 11, creating indels that disrupt repressor binding and increase gamma globin expression. The editing components are introduced into the target cell population as a ribonucleoprotein complex by electroporation. 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 ≥70% CD34/CD45+ purity and ≥70% on-target editing.

EDIT-301 CD34+ hematopoietic stem cells

RENIZGAMGLOGENE AUTOGEDTEMCEL [USAN]

Renizgamglogene autogedtemcel [WHO-DD]

renizgamglogene autogedtemcel [INN]