Alvamemugene Sulseparvovec

Approval Year

Substance Class	Structurally Diverse Created by `admin` on `Sat Dec 16 18:06:14 GMT 2023` Edited by `admin` on `Sat Dec 16 18:06:14 GMT 2023`
Source Materials Class	ORGANISM
Source Materials Type	VIRUS
Source Materials State	LIVE NON-REPLICATING GENETICALLY MODIFIED
Organism Family	Parvoviridae
Organism Genus	Dependoparvovirus
Organism Species	adeno associated virus
Infraspecific Type	SUBSTRAIN
Infraspecific Name	rAAV-LK03
Part	whole
Record UNII	HUH56XF3P7
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Alvamemugene Sulseparvovec

Official Name

English

DNA (recombinant human adeno-associated virus AAV-LK03 vector LB-001 human methylmalonyl-coenzyme A mutase codon-optimized transgene plus porcine teschovirus 1 peptide 2A plus human albumin homology arms plus human adeno-associated virus 2 inverted termi

Common Name

English

alvamemugene sulseparvovec [INN]

Common Name

English

recombinant, non-replicating adeno-associated virus (rAAV) vector with a liver-trophic LK03 capsid, encoding codon-optimised human methylmalonyl-CoA mutase (MMUT, also referred to as MUT), preceded by the self-cleaving 2A-peptide of porcine teschovirus 1

Common Name

English

Code System Code Type Description

INN

12220

Created by admin on Sat Dec 16 18:06:14 GMT 2023 , Edited by admin on Sat Dec 16 18:06:14 GMT 2023

PRIMARY

CAS

2640356-62-1

Created by admin on Sat Dec 16 18:06:14 GMT 2023 , Edited by admin on Sat Dec 16 18:06:14 GMT 2023

PRIMARY

NCI_THESAURUS

C190453

Created by admin on Sat Dec 16 18:06:14 GMT 2023 , Edited by admin on Sat Dec 16 18:06:14 GMT 2023

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FDA UNII

HUH56XF3P7

Created by admin on Sat Dec 16 18:06:14 GMT 2023 , Edited by admin on Sat Dec 16 18:06:14 GMT 2023

PRIMARY

Related Record Type Details


					47VA6JGH79

METHYLMALONYL-COA MUTASE, MITOCHONDRIAL

EXPRESSED PROTEIN->VECTOR

Amount:

Related Record Type Details


					HUH56XF3P7

Alvamemugene Sulseparvovec

ACTIVE MOIETY

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
TRANSGENE					Alvamemugene sulseparvovec transgene insert	3CLT8HFQ3Q