Rebisufligene Etisparvovec

Investigational

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Wed Apr 02 06:17:37 GMT 2025` Edited by `admin` on `Wed Apr 02 06:17:37 GMT 2025`
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	SUBSTRAIN
Infraspecific Name	scAAV9.U1a.hSGSH
Part	whole
Record UNII	V28L3Q0LVZ
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

rebisufligene etisparvovec [INN]

Preferred Name

English

Rebisufligene Etisparvovec

Official Name

English

DNA (synthetic adeno-associated virus 9 vector scAAV9.U1a.hSGSH human N-Sulfoglucosamine sulfohydrolase-specifying)

Common Name

English

UX111

Code

English

ABO-102

Common Name

English

SCAAV9.U1A.HSGSH

Code

English

Non-replicating, self-complementary, and recombinant adeno-associated virus (AAV) serotype 9 viral vector encoding a functional variant of the human N-sulfoglucosamine sulfohydrolase (hSGSH) transgene

Common Name

English

UX-111

Code

English

REBISUFLIGENE ETISPARVOVEC [USAN]

Common Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

433714