NULABEGLOGENE AUTOGEDTEMCEL
UNII H9VA9H989B

Substance Identification & Data

This profile provides standardized clinical and technical data for Nulabeglogene Autogedtemcel, uniquely identified by the FDA Unique Ingredient Identifier (UNII) H9VA9H989B.

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

H9VA9H989B

CAS Registry Number

N/A

RxNorm Concept ID

N/A

Detailed Substance Profile

Preferred Name

NULABEGLOGENE AUTOGEDTEMCEL

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

NCI Thesaurus

C190451

National Cancer Institute reference terminology for clinical and research data.

INN ID

12025

Sequential identifier assigned via the WHO International Nonproprietary Name program.

USAN ID

LM-06

Identifier assigned by the United States Adopted Names Council.

Substance Type

Nulabeglogene 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 Nulabeglogene 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

Nulabeglogene Autogedtemcel

Common Names & Synonyms

Autologous, CRISPR/Cas9-based, gene-editing hematopoietic stem cell therapy (HSCT) targeted at the beta-globin (HBB) gene and designed to correct its mutation responsible for sickle cell disease (that is, designed to convert the mutant HbS allele of the HBB gene to its HbA allele), thereby restoring normal hemoglobin levels

NULABEGLOGENE AUTOGEDTEMCEL [USAN]

autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) obtained by apheresis from sickle cell patients, genetically modified ex vivo by CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) mediated gene editing consisting of a single guide RNA (sgRNA) targeting the first exon of the human β-globin (HBB) gene, and using a homology-directed repair mechanism to correct the nucleic acid sequence encoding the glutamic acid to valine mutation at position 6 in the HBB protein via an adeno-associated virus serotype 6 (AAV6) vector in which the sgRNA targeting sequence is located between two HBB homology arms. Following genetic modification, each cell can have one of six combinations of HBB allele (WT/WT, INDEL/WT, INDEL/INDEL, INDEL/HR, WT/HR, HR/HR, where WT refers to the wild type sickle cell disease allele, INDEL refers to an insertion/deletion event but no correction of the HBB gene, and HR refers to a corrected HBB allele). On average 56% of the cells have at least one corrected HBB allele (HR). The cell suspension is enriched for CD34+ cells using magnetic bead separation. The substance consists of cells with the CD45+ and CD34+ phenotype, with ≥70% CD34 purity. The functional characterization of the cells is based on the ability to form erythroid and myeloid colonies on semisolid methylcellulose-based medium.

nulabeglogene autogedtemcel [INN]

Technical Codes

GPH-101

GPH101

External Clinical & Regulatory Resources

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