UNII 4G7DT7PP3L

Unique Ingredient Identifier: 4G7DT7PP3L

The Unique Ingredient Identifier (UNII) is a unique alphanumeric code assigned to a specific substance or ingredient by the FDA (Food and Drug Administration). UNII codes are non-proprietary, free, unique, unambiguous and nonsemantic. Each UNII code consists of 10 characters with both letters and numbers. The first two characters represent the source of the substance while the remaining eight characters represent the specific substance. UNII codes are primarily used to identify and track substances in several drug databases and regulatory documents related to drugs, biologics, and medical devices.

Preferred Substance Name: BRINRETIGENE VESGEDPARVOVEC

The Preferred Substance Name (PSN) is the official or preferred name of a substance or ingredient. The preferred name provides a standardized and consistent name for each substance is used in the UNII code system. The preferred name may differ from other names also commonly used to refer the same substance. The PSN is usually the name used by regulatory agencies like the FDA.

Registry Number: 2768756-11-0

The Registry Number is the unique numerical identifiers assigned to a substance or ingredient by the Chemical Abstracts Service (CAS). The CAS registry number is a used and recognized standard identifier by many industries and regulatory agencies. The Registry Number facilitates cross-referencing substances between different databases that may use different naming conventions or identifiers.

NCI Thesaurus Concept Code: C203111

The NCI Thesaurus give reference terminology for many systems. The code includes vocabulary for clinical care, translational research, basic research, public information and administrative activities.

INN ID: 12734

Sequential number assigned by World Health Organization's International Nonproprietary Name (INN).

Substance Type: structurallyDiverse

The International Organization for Standardization - ISO 11238 substance type is used as a data element in the UNII code system to provide additional information about the type of substance being identified.

The following synonyms and mappings provide alternative ways to refer to the same substance or ingredient. Synonyms may include different names or spellings, alternate chemical names, trade names, or other common names.

• BRINRETIGENE VESGEDPARVOVEC - Name identified as having official status
• BRINRETIGENE VESGEDPARVOVEC [INN] - Common Name
• 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) - Common Name
• EDIT-101 - Code
• 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 - Common Name

Common Chemistry

CAS Common Chemistry is an open community resource of the American Chemical Society for accessing chemical information. The chemical information in the CAS database is curated by expert scientists and includes information of common and frequently regulated chemicals and information applicable to high school and undergraduate chemistry classes.

Inxight Drugs

The National Center for Advancing Translational Sciences (NCATS) Inxight Drugs database has a comprehensive drug development information. The NCATS Inxight Drugs includes manually curated data provided by private companies and FDA. The database has marketing and regulatory status information, rigorous drug ingredient definitions, biological activity, clinical use, etc.

DailyMed Regulated Products

The National Library of Medicine DailyMed is a searchable database that includes the most recent product labeling information submitted to the FDA by private companies.

NCATS GSRS Full Record

The Global Substance Registration System (GSRS) was designed to facilitate global monitoring of human and animal medicinal, food, tobacco, and cosmetic products. GSRS includes unique substance identifiers consistent with the ISO 11238 standard.

NCI Thesaurus

NCI Thesaurus (NCIt) by the National Cancer Institute provides reference terminology for clinical care, translational and basic research, and public information and administrative activities.