Arvenacogene sanparvovec transgene insert

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Wed Apr 02 19:53:09 GMT 2025` Edited by `admin` on `Wed Apr 02 19:53:09 GMT 2025`
Nucleic Acid Type	VECTOR
Sequence Origin	VIRAL, HUMAN
Sequence Type	COMPLETE
Record UNII	X96BYJ9M32
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

Arvenacogene sanparvovec transgene insert

Common Name

English

DNA (recombinant adeno-associated virus vector pAAV-hFIXco-v2 Mus musculus transtyretin gene promoter plus human ?-globin gene intron fragment plus human preproblood coagulation factor IX [384-leucine] codon-optimized gene plus human somatotropin gene po

Preferred Name

English

Code System Code Type Description

CAS

2829392-57-4

Created by admin on Wed Apr 02 19:53:09 GMT 2025 , Edited by admin on Wed Apr 02 19:53:09 GMT 2025

PRIMARY

FDA UNII

X96BYJ9M32

Created by admin on Wed Apr 02 19:53:09 GMT 2025 , Edited by admin on Wed Apr 02 19:53:09 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
Deoxyribose (DNA)	1_1-1_3123	3123 / 3123

Linkage	Site Range	Site Count
Phosphate	1_2-1_3123	3122 / 3122

Name	Property Type	Amount	Referenced Substance	Defining	Parameters	References
hBGintron	NUCLEIC ACID FEATURE

L_ITR2	NUCLEIC ACID FEATURE

hFIX_R338L	NUCLEIC ACID FEATURE

hGHpolyA	NUCLEIC ACID FEATURE

prmTTR	NUCLEIC ACID FEATURE

R_ITR2	NUCLEIC ACID FEATURE