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

Approval Year

Substance Class Structurally Diverse
Created
by admin
on Sat Dec 16 15:15:21 GMT 2023
Edited
by admin
on Sat Dec 16 15:15:21 GMT 2023
Source Materials Class ORGANISM
Source Materials Type AUTOLOGOUS HUMAN
Source Materials State LIVE GENETICALLY MODIFIED
Source Materials Parent
Part bone-marrow-and-peripheral-blood
Fraction Material Type CELLS
Fraction Name hematopoietic stem and progenitor cells
Record UNII
MEE8487RTP
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
BETIBEGLOGENE AUTOTEMCEL
USAN   INN  
Official Name English
AUTOLOGOUS CD34+ CELLS ENCODING .BETA.A-T87Q-GLOBIN GENE
Common Name English
BB-305
Code English
ZYNTEGLO
Brand Name English
betibeglogene autotemcel [INN]
Common Name English
AUTOLOGOUS CD34+ ENRICHED CELL POPULATION THAT CONTAINS HEMATOPOIETIC STEM CELLS (HSC'S) TRANSDUCED WITH THE LENTIGLOBIN BB305 LENTIVIRAL VECTOR (LVV) ENCODING FOR THE .BETA.A-T87Q-GLOBIN GENE. THE CD34+ ENRICHED CELL POPULATION CONTAINING HEMATOPOIETIC
Common Name English
Betibeglogene autotemcel [WHO-DD]
Common Name English
LENTIGLOBIN BB305
Code English
BETIBEGLOGENE AUTOTEMCEL [USAN]
Common Name English
Classification Tree Code System Code
FDA ORPHAN DRUG 390513
Created by admin on Sat Dec 16 15:15:21 GMT 2023 , Edited by admin on Sat Dec 16 15:15:21 GMT 2023
Code System Code Type Description
DAILYMED
MEE8487RTP
Created by admin on Sat Dec 16 15:15:21 GMT 2023 , Edited by admin on Sat Dec 16 15:15:21 GMT 2023
PRIMARY
INN
10900
Created by admin on Sat Dec 16 15:15:21 GMT 2023 , Edited by admin on Sat Dec 16 15:15:21 GMT 2023
PRIMARY
USAN
GH-155
Created by admin on Sat Dec 16 15:15:21 GMT 2023 , Edited by admin on Sat Dec 16 15:15:21 GMT 2023
PRIMARY
FDA UNII
MEE8487RTP
Created by admin on Sat Dec 16 15:15:21 GMT 2023 , Edited by admin on Sat Dec 16 15:15:21 GMT 2023
PRIMARY
WIKIPEDIA
Zynteglo
Created by admin on Sat Dec 16 15:15:21 GMT 2023 , Edited by admin on Sat Dec 16 15:15:21 GMT 2023
PRIMARY
RXCUI
2610349
Created by admin on Sat Dec 16 15:15:21 GMT 2023 , Edited by admin on Sat Dec 16 15:15:21 GMT 2023
PRIMARY
NCI_THESAURUS
C123881
Created by admin on Sat Dec 16 15:15:21 GMT 2023 , Edited by admin on Sat Dec 16 15:15:21 GMT 2023
PRIMARY
Related Record Type Details
EXPRESSED PROTEIN->VECTOR
Related Record Type Details
ACTIVE MOIETY

Agent Modifications

Modification Process Modification Role Modification Type Amount Fragment Name Fragment ID
CD34+ Enrichment CELL SEPARATION
LENTIVIRAL TRANSFECTION BETIBEGLOGENE DAROLENTIVEC 7VEJ4HT24J
CD34+ Cell Labeling IMMUNOLOGICAL

Structural Modifications

Modification Type Location Site Location Type Residue Modified Extent Fragment Name Fragment Approval
VECTOR EXPRESSED PROTEIN UNSPECIFIED SUBSTANCE
f4353dfd
(not in database)
INSERTED GENE EXPRESSION VECTOR UNSPECIFIED SUBSTANCE
53ad4970
(not in database)
Name Property Type Amount Referenced Substance Defining Parameters References
ANTIGEN EXPRESSED BIOLOGICAL