Brinretigene Vesgedparvovec

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Sat Dec 16 19:59:39 GMT 2023` Edited by `admin` on `Sat Dec 16 19:59:39 GMT 2023`
Source Materials Class	ORGANISM
Source Materials Type	RECOMBINANT VIRUS
Source Materials State	LIVE NON-REPLICATING GENETICALLY MODIFIED
Organism Family	Parvoviridae
Organism Genus	Dependoparvovirus
Organism Species	adeno-associated dependoparvovirus a
Infraspecific Type	RECOMBINANT VECTOR
Infraspecific Name	AAV5/AAV2-U6-CEP290gRNA323/64-GRK1-SaCas9
Part	whole
Record UNII	4G7DT7PP3L
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Brinretigene Vesgedparvovec

Official Name

English

EDIT-101

Code

English

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

Common Name

English

brinretigene vesgedparvovec [INN]

Common Name

English

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 (SV

Common Name

English

Code System Code Type Description

FDA UNII

4G7DT7PP3L

Created by admin on Sat Dec 16 19:59:40 GMT 2023 , Edited by admin on Sat Dec 16 19:59:40 GMT 2023

PRIMARY

INN

12734

Created by admin on Sat Dec 16 19:59:40 GMT 2023 , Edited by admin on Sat Dec 16 19:59:40 GMT 2023

PRIMARY

CAS

2768756-11-0

Created by admin on Sat Dec 16 19:59:40 GMT 2023 , Edited by admin on Sat Dec 16 19:59:40 GMT 2023

PRIMARY

Related Record Type Details


					P8FJZ7FHB8

CRISPR-associated endonuclease Cas9

EXPRESSED PROTEIN->VECTOR

Amount:


					D2W9FX5RB3

Centrosomal protein of 290 kDa

TARGET->ENZYME REPLACEMENT

Amount:

Related Record Type Details


					4G7DT7PP3L

Brinretigene Vesgedparvovec

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
INSERTED GENE EXPRESSION VECTOR					Brinretigene vesgedparvovec transgene insert	Z9F4EN4DQZ