AZAMIDUGENE AUTOTEMCEL

UNII:	7BAL95SK9C
Type:	INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:	AZAMIDUGENE AUTOTEMCEL
GSRS Full Record:	7BAL95SK9C i

AUTOLOGOUS CD34+ CELLS OBTAINED FROM MOBILISED PERIPHERAL BLOOD, TRANSDUCED WITH A SELF-INACTIVATING LENTIVIRAL VECTOR EXPRESSING HUMAN ALPHA-L-IDURONIDASE (IDUA) UNDER THE CONTROL OF A HUMAN PHOSPHOGLYCERATE KINASE (PGK) PROMOTER THE CONSTRUCT IS FLANKED BY 5' AND 3' LONG TERMINAL REPEATS (LTRS) AND ALSO CONTAINS A HIV-1 .PSI. PACKAGING SIGNAL, A REV RESPONSE ELEMENT (RRE), A CENTRAL POLYPURINE TRACT (CPPT) SEQUENCE AND THE WOODCHUCK HEPATITIS VIRUS POST-TRANSCRIPTIONAL REGULATORY ELEMENT (WPRE). THE VECTOR IS PSEUDOTYPED WITH THE ENVELOPE GLYCOPROTEIN (G) OF THE VESICULAR STOMATITIS VIRUS (VSV). THE CD34+ CELL POPULATION WAS ENRICHED USING IMMUNOMAGNETIC MICROBEADS, AND THE CELLS CULTURED IN THE PRESENCE OF MEDIA CONTAINING FMSRELATED TYROSINE KINASE 3 LIGAND (FLT-3L), INTERLEUKIN 3 (IL-3), THROMBOPOIETIN (TPO) AND STEM CELL FACTOR (SCF). THE FINAL SUBSTANCE CONTAINS 70% CD34+ CELLS
AZAMIDUGENE AUTOTEMCEL [USAN]
Autologous CD34+ cells obtained from mobilized peripheral blood, transduced with a self-inactivating lentiviral vector expressing human alpha-L-iduronidase (IDUA) under the control of a human phosphoglycerate kinase (PGK) promoter
Azamidugene Autotemcel
OTL-203
azamidugene autotemcel [INN]

Note

UNIIs are generated based on scientific identity characteristics using ISO 11238 data elements. UNII availability does not imply any regulatory review or approval. Synonyms and mappings are based on the best public information available at the time of publication. Please report any problems/errors associated with this data to FDA-SRS@fda.hhs.gov.

AZAMIDUGENE AUTOTEMCEL

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