Lonimecgene Renparvovec

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Wed Apr 02 19:41:41 GMT 2025` Edited by `admin` on `Wed Apr 02 19:41:41 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	RECOMBINANT VECTOR
Infraspecific Name	scAAV9-MeP426-miniMECP2-miRARE
Part	whole
Record UNII	NVK87P6NXW
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

TSHA-102

Preferred Name

English

Lonimecgene Renparvovec

Official Name

English

LONIMECGENE RENPARVOVEC [USAN]

Common Name

English

recombinant, self-complementary (dimeric), non-replicating adeno-associated virus serotype 9 (scAAV9) vector encoding a miniaturised form of human methyl-CpG binding protein 2 (MECP2) containing the methyl-CpG-binding domain (MBD) and the NCoR/SMRT (sile

Common Name

English

lonimecgene renparvovec [INN]

Common Name

English

DNA (self-complementary adeno-associated virus 9 vector TSHA-102 mouse neuronal-specific promoter MeP426 variant plus human gene MECP2 fragment plus microRNA-binding element RDH1pA plus miRARE (miRNA responsive auto-regulatory element) plus human gene ME

Common Name

English

Code System Code Type Description

SMS_ID

300000032082