U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Approval Year

Unknown
149124
Name Type Language
BMN 307 (AN AAV5-PHENYLALANINE HYDROXYLASE (PAH) GENE THERAPY)
Preferred Name English
DOMOFENOGENE ZALFAPARVOVEC
INN  
Official Name English
DNA (RECOMBINANT REPLICATION-DEFICIENT ADENO-ASSOCIATED VIRUS 5 VECTOR BMN 307 HUMAN LIVER-SPECIFIC TRANSCRIPTION REGULATORY ELEMENT PLUS HUMAN PHENYLALANINE 4-HYDROXYLASE CODON-OPTIMIZED GENE HPAH PLUS ADENO-ASSOCIATED VIRUS 2 INVERTED TERMINAL REPEAT F
Common Name English
domofenogene zalfaparvovec [INN]
Common Name English
RECOMBINANT, REPLICATION-INCOMPETENT ADENOASSOCIATED VIRUS SEROTYPE 5 (AAV5) VECTOR ENCODING CODON-OPTIMIZED HUMAN PHENYLALANINE HYDROXYLASE (HPAH) UNDER CONTROL OF A LIVER-SELECTIVE TRANSCRIPTION REGULATORY ELEMENT (TRE), CONSISTING OF THE APOLIPOPROTEI
Common Name English
BMN-307
Common Name English
Code System Code Type Description
CAS
2382896-04-8
Created by admin on Wed Apr 02 07:09:43 GMT 2025 , Edited by admin on Wed Apr 02 07:09:43 GMT 2025
PRIMARY
SMS_ID
300000000720
Created by admin on Wed Apr 02 07:09:43 GMT 2025 , Edited by admin on Wed Apr 02 07:09:43 GMT 2025
PRIMARY
NCI_THESAURUS
C184853
Created by admin on Wed Apr 02 07:09:43 GMT 2025 , Edited by admin on Wed Apr 02 07:09:43 GMT 2025
PRIMARY
INN
11598
Created by admin on Wed Apr 02 07:09:43 GMT 2025 , Edited by admin on Wed Apr 02 07:09:43 GMT 2025
PRIMARY
FDA UNII
PN77BRS6J9
Created by admin on Wed Apr 02 07:09:43 GMT 2025 , Edited by admin on Wed Apr 02 07:09:43 GMT 2025
PRIMARY
NIH
NCATS
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