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

Approval Year

Substance Class Nucleic Acid
Created
by admin
on Wed Apr 02 11:13:03 GMT 2025
Edited
by admin
on Wed Apr 02 11:13:03 GMT 2025
Nucleic Acid Type VECTOR
Sequence Type COMPLETE
Record UNII
KND6MY3R59
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
NULABEGLOGENE AUTOGEDTEMCEL TRANSGENE INSERT
Common Name English
NULABEGLOGENE AUTOGEDTEMCEL TRANSGENE INSERT ENCODING HUMAN HBB
Preferred Name English
Code System Code Type Description
FDA UNII
KND6MY3R59
Created by admin on Wed Apr 02 11:13:03 GMT 2025 , Edited by admin on Wed Apr 02 11:13:03 GMT 2025
PRIMARY
Sugar Site Range Site Count
Deoxyribose (DNA) 1_1-1_2700 2700 / 2700
Linkage Site Range Site Count
Phosphate 1_2-1_2700 2699 / 2699
Name Property Type Amount Referenced Substance Defining Parameters References
3’ ITR NUCLEIC ACID FEATURE
GAG (Glutamate codon to correct the valine at this ) NUCLEIC ACID FEATURE
R-02(Mutated-guide RNA homologous target sequence) NUCLEIC ACID FEATURE
Homology arm upstream of the R-02 sequence NUCLEIC ACID FEATURE
Haemoglobin subunit beta NUCLEIC ACID FEATURE
5’-ITR NUCLEIC ACID FEATURE
Homology arm downstream of the R-02 sequence-HA-3' NUCLEIC ACID FEATURE
ATG site (Haemoglobin translation initiation site) NUCLEIC ACID FEATURE