BRINRETIGENE VESGEDPARVOVEC
UNII 4G7DT7PP3L

Substance Identification & Data

This profile provides standardized clinical and technical data for Brinretigene Vesgedparvovec, uniquely identified by the FDA Unique Ingredient Identifier (UNII) 4G7DT7PP3L.

Technical mappings include the Chemical Abstracts Service (CAS) Registry Number 2768756-11-0 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

4G7DT7PP3L

CAS Registry Number

2768756-11-0

RxNorm Concept ID

N/A

Detailed Substance Profile

Preferred Name

BRINRETIGENE VESGEDPARVOVEC

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

NCI Thesaurus

C203111

National Cancer Institute reference terminology for clinical and research data.

INN ID

12734

Sequential identifier assigned via the WHO International Nonproprietary Name program.

Substance Type

Brinretigene Vesgedparvovec

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

Synonyms and Nomenclature

This section provides a complete list of nomenclature and identifier mappings for Brinretigene Vesgedparvovec. 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

Brinretigene Vesgedparvovec

Common Names & Synonyms

Brinretigene vesgedparvovec [WHO-DD]

DNA (recombinant adeno-associated virus vector EDIT-101 human U6 promoter plus nephrocystin 6 intron 26-specific CEP290-323 guide RNA gene plus human U6 promoter plus nephrocystin 6 intron 26-specific CEP290-64 guide RNA gene plus human rhodopsin kinase promoter plus simian virus 40 nuclear localization signal peptide fusion protein with Staphylococcus aureus CRISPR-associated endodeoxyribonuclease CAS9 fusion protein with simian virus 40 nuclear localization signal peptide gene plus synthetic polyadenylation signal plus adeno-associated virus 2 inverted terminal repeat flanks-specifying)

Recombinant, non-replicating adeno-associated virus serotype 5 (rAAV5) vector encoding (1) codon-optimized Staphylococcus aureus clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) flanked by simian virus 40 (SV40) nuclear localisation signal peptide sequences and (2) two guide RNAs (gRNA-323 and gRNA-64) targeting intron 26 of the human centrosomal protein 290 (CEP290, nephrocystin-6) gene. Expression of the CRISPR Cas9 gene is under control of the human G protein-coupled receptor kinase 1 (GRK1, rhodopsin kinase) promoter along with an SV40 splice donor/splice acceptor site, a Kozak sequence and a small synthetic polyadenylation signal. Expression of the two gRNAs is each under control of a human small nuclear RNA U6 polymerase III promoter. The vector genome is flanked by adeno-associated virus 2 (AAV2) inverted terminal repeats (ITRs). Expression of the gene editing components is intended to remove or invert the mutation-containing intronic region of the CEP290 gene (IVS26), and to restore functional CEP290 protein levels

brinretigene vesgedparvovec [INN]

Technical Codes

EDIT-101

External Clinical & Regulatory Resources

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