NENZINACOGENE AUTOGELEUCEL
UNII 2TZV23CKJ9

Substance Identification & Data

This profile provides standardized clinical and technical data for Nenzinacogene Autogeleucel, uniquely identified by the FDA Unique Ingredient Identifier (UNII) 2TZV23CKJ9.

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

2TZV23CKJ9

CAS Registry Number

N/A

RxNorm Concept ID

N/A

Detailed Substance Profile

Preferred Name

NENZINACOGENE AUTOGELEUCEL

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

INN ID

13868

Sequential identifier assigned via the WHO International Nonproprietary Name program.

Substance Type

Nenzinacogene autogeleucel

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 Nenzinacogene Autogeleucel. 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

Nenzinacogene autogeleucel

Common Names & Synonyms

autologous (CD19+) B lymphocytes obtained from peripheral blood via leukapheresis, electroporated with a CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9) /guide RNA (gRNA) nucleoprotein complex to disrupt the C-C chemokine receptor type 5 (CCR5) gene locus (targeting exon 2). The cells are then transduced with a recombinant adeno-associated virus serotype 6 vector (rAAV6) to introduce a human coagulation factor IX (FIX) Padua/Malmö variant, expression cassette to the CCR5 locus by homology-directed repair. The integrated sequence comprises a 5' CCR5 homology arm, the transgene expression cassette and a 3' CCR5 homology arm. The transgene expression cassette comprises the MND promoter, a Kozak sequence, a codon-optimized, CG-depleted human FIX [Padua (R338L) and Malmö (T148A) variants] coding sequence, followed by a bovine growth hormone polyadenylation (polyA) sequence. The leukapheresis material is enriched for B lymphocytes with CD19+ beads. The cells are cultured and activated with human CD40 ligand and cytosine-phosphate-guanine (CpG) dinucleotides in the presence of interleukin 21 (IL-21). Following vector transduction, the cells are subsequently expanded and cultured in media containing cytokines (IL-2, IL-6, IL-10, IL-15) to induce early-stage differentiation, followed by culture in media containing only IL-6, IL-15, and IFNα-2β for late-stage differentiation. The final cells secrete FIX and consist primarily of lymphocytes (>80% CD38+), with >25% on target F9 transgene insertion, and with less than 2% non-B lymphocyte lineage cells

nenzinacogene autogeleucel [INN]

External Clinical & Regulatory Resources

Previous UNII2TZH7KB8N8

Next UNII2U0001Z916