NEFONADOGENE VONZUPARVOVEC
UNII FBB99Y9WCY

Substance Identification & Data

This profile provides standardized clinical and technical data for Nefonadogene Vonzuparvovec, uniquely identified by the FDA Unique Ingredient Identifier (UNII) FBB99Y9WCY.

Technical mappings include the Chemical Abstracts Service (CAS) Registry Number 2779554-99-1 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

FBB99Y9WCY

CAS Registry Number

2779554-99-1

RxNorm Concept ID

N/A

Detailed Substance Profile

Preferred Name

NEFONADOGENE VONZUPARVOVEC

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

NCI Thesaurus

C211770

National Cancer Institute reference terminology for clinical and research data.

INN ID

12868

Sequential identifier assigned via the WHO International Nonproprietary Name program.

Substance Type

structurallyDiverse

ISO 11238 classification category (e.g., Chemical, Polymer, Protein).

Synonyms and Nomenclature

This section provides a complete list of nomenclature and identifier mappings for Nefonadogene Vonzuparvovec. 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

NEFONADOGENE VONZUPARVOVEC

Common Names & Synonyms

DNA (RECOMBINANT REPLICATION-DEFECTIVE ADENO-ASSOCIATED VIRUS 2 VECTOR NFS02 HUMAN CYTOMEGALOVIRUS ENHANCER PLUS HUMAN CYTOMEGALOVIRUS PROMOTER PLUS MACACINE HERPESVIRUS 3 CHIMERIC INTRON PLUS HUMAN HEME A FARNESYLTRANSFERASE GENE COX10 MITOCHONDRIAL TARGETING SIGNAL PEPTIDE FUSION WITH HUMAN MITOCHONDRIA NADH DEHYDROGENASE SUBUNIT 1 CODON-OPTIMIZED GENE PLUS HEME A FARNESYLTRANSFERASE GENE COX10 3'-UNTRANSLATED REGION PLUS SIMIAN VIRUS 40 POLYADENYLATION SIGNAL PLUS ADENO-ASSOCIATED VIRUS 2 INVERTED TERMINAL REPEAT FLANKS-SPECIFYING)

NEFONADOGENE VONZUPARVOVEC [INN]

RECOMBINANT, NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 2 (RAAV2) VECTOR, ENCODING A MITOCHONDRIAL TARGETING SEQUENCE FROM THE HUMAN HEME A FARNESYLTRANSFERASE GENE (COX10) FUSED TO CODON-OPTIMISED HUMAN MITOCHONDRIAL NADH DEHYDROGENASE SUBUNIT 1 (MT-ND1, NADH1, ND1; ALSO KNOWN AS NAD-HUBIQUINONE OXIDOREDUCTASE CHAIN 1), UNDER CONTROL OF THE CYTOMEGALOVIRUS (CMV) IMMEDIATE EARLY ENHANCER/PROMOTER AND THE SIMIAN VIRUS 40 (SV40) POLYADENYLATION SIGNAL. THE 5' UNTRANSLATED REGION (UTR) ALSO CONTAINS A MACACINE HERPESVIRUS 3 CHIMERIC INTRON, 3' OF THE CMV ENHANCER/PROMOTER AND THE KOZAK SEQUENCE, AND THE 3' UNTRANSLATED REGION COMPRISES THE COX10 3' UTR. THE EXPRESSION CASSETTE IS FLANKED BY AAV2 INVERTED TERMINAL REPEATS

External Clinical & Regulatory Resources

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