UNII X9AT6SDG44

Unique Ingredient Identifier: X9AT6SDG44

The Unique Ingredient Identifier (UNII) is a unique alphanumeric code assigned to a specific substance or ingredient by the FDA (Food and Drug Administration). UNII codes are non-proprietary, free, unique, unambiguous and nonsemantic. Each UNII code consists of 10 characters with both letters and numbers. The first two characters represent the source of the substance while the remaining eight characters represent the specific substance. UNII codes are primarily used to identify and track substances in several drug databases and regulatory documents related to drugs, biologics, and medical devices.

Preferred Substance Name: ATAGLOGENE AUTOGETEMCEL

The Preferred Substance Name (PSN) is the official or preferred name of a substance or ingredient. The preferred name provides a standardized and consistent name for each substance is used in the UNII code system. The preferred name may differ from other names also commonly used to refer the same substance. The PSN is usually the name used by regulatory agencies like the FDA.

ITIS TSN: 180092

The Taxonomic Serial Number is a unique identifier for a specific species of organism in the Integrated Taxonomic Information System. The ITIS is a comprehensive database of taxonomic information on plants, animals, fungi, and microbes, maintained by a partnership of several US government agencies.

NCBI: 9606

The NCBI taxonomy organism ID is a unique numeric identifier used to specify species of organisms by the National Center for Biotechnology Information (NCBI) Taxonomy database.

INN ID: 13584

Sequential number assigned by World Health Organization's International Nonproprietary Name (INN).

Substance Type: structurallyDiverse

The International Organization for Standardization - ISO 11238 substance type is used as a data element in the UNII code system to provide additional information about the type of substance being identified.

The following synonyms and mappings provide alternative ways to refer to the same substance or ingredient. Synonyms may include different names or spellings, alternate chemical names, trade names, or other common names.

• ATAGLOGENE AUTOGETEMCEL - Name identified as having official status
• ATAGLOGENE AUTOGETEMCEL [INN] - Common Name
• AUTOLOGOUS CD34+ HEMATOPOIETIC STEM CELLS OBTAINED BY APHERESIS, ELECTROPORATED WITH A MULTI-COMPONENT CRISPR/CAS9 (CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS/CRISPR-ASSOCIATED PROTEIN 9)-GUIDE RNA (GRNA) TRANSFORMER BASE EDITOR NUCLEOPROTEIN COMPLEX THAT TARGETS THE PROMOTER REGIONS OF THE TWO .GAMMA.-GLOBIN GENES (HBG1 AND HBG2) TO DISRUPT BINDING OF THE TRANSCRIPTIONAL REPRESSOR BCL11A (B-CELL LYMPHOMA/LEUKEMIA 11A), LEADING TO THE RE-EXPRESSION OF .GAMMA.-GLOBIN AND INCREASED PRODUCTION OF FOETAL HAEMOGLOBIN (HBF). THE TRANSFORMER BASE EDITING SYSTEM COMPRISES TWO MRNAS AND TWO SINGLE GUIDE RNAS. THE FIRST MRNA (MRNA-L) ENCODES AN IMPAIRED FORM OF THE STREPTOCOCCUS PYOGENES CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS (CRISPR) ASSOCIATED PROTEIN 9 AND IS REFERRED TO AS CAS9 NICKASE D10A. THE SECOND MRNA (MRNA-EK) ENCODES THREE INDEPENDENT PROTEINS, SEPARATED BY 2A PEPTIDES; THE FIRST PROTEIN (MCP-UGI-MA3CDA1-MA3DCDI), IS A FUSION PROTEIN COMPRISING (5' TO 3') AN RNA APTAMER BINDING PROTEIN (BACTERIOPHAGE MS2 COAT PROTEIN (MCP)), A URACIL DNA GLYCOSYLASE INHIBITOR (UGI), THE MOUSE APOLIPOPROTEIN B MRNA-EDITING COMPLEX 3 (APOBEC3, MA3) CYTIDINE DEAMINASE DOMAIN 1 (MA3CDA1), A TOBACCO ETCH VIRUS (TEV) PROTEASE CLEAVAGE SITE, AND THE MOUSE APOBEC3 DEOXYCYTIDINE DEAMINASE INHIBITOR DOMAIN (DCDI) (MA3DCDI). THE SECOND PROTEIN (N22P-TEVC) CONTAINS AN RNA APTAMER BINDING PROTEIN (BACTERIOPHAGE N PEPTIDE 1-22) FUSED WITH THE C-TERMINAL FRAGMENT OF THE TOBACCO ETCH VIRUS PROTEASE (TEVC). THE THIRD PROTEIN (TEVN) COMPRISES THE N-TERMINAL FRAGMENT OF THE TEV PROTEASE. THE FIRST SINGLE GUIDE RNA IS A HELPER GRNA (HSGRNA) THAT GUIDES NCAS9 TO THE FIRST TARGET SITE AND CONTAINS AN MS2 HAIRPIN TO RECRUIT THE MCP-UGI-MA3CDA1-MA3DCDI FUSION PROTEIN. THE SECOND SGRNA IS A MODIFYING GRNA (MSGRNA) THAT GUIDES A SECOND NCAS9 TO THE GENE EDITING TARGET SITE AND CONTAINS BOX B HAIRPINS TO RECRUIT THE N22P-TEVC PROTEIN. WHEN THE TWO GUIDE RNAS BIND ON-TARGET, THE TWO HALVES OF THE TEV PROTEASE (TEVN AND TEVC) RECONSTITUTE, AND THE PROTEASE CAN CLEAVE OFF THE DEOXYCYTIDINE DEAMINASE INHIBITOR (MA3DCDI) FROM THE MRNA EDITING CYTIDINE DEAMINASE DOMAINE 1 (MA3CDA1) TO EDIT THE TRANSCRIPTIONAL REPRESSOR BCL11A BINDING DOMAIN OF THE .GAMMA.-GLOBIN PROMOTER REGIONS. THE APHERESIS MATERIAL IS ENRICHED IN CD34+ CELLS USING IMMUNOMAGNETIC MICROBEADS, GENE-EDITED AND THEN CULTURED IN THE PRESENCE OF THROMBOPOIETIN (TPO), STEM CELL FACTOR (SCF) AND FMS-LIKE TYROSINE KINASE 3 LIGAND (FLT-3L). THE FINAL SUBSTANCE CONTAINS >=90% CD34+ CELLS AND HAS A TARGET GENE EDITING EFFICIENCY OF >=30% - Common Name

Inxight Drugs

The National Center for Advancing Translational Sciences (NCATS) Inxight Drugs database has a comprehensive drug development information. The NCATS Inxight Drugs includes manually curated data provided by private companies and FDA. The database has marketing and regulatory status information, rigorous drug ingredient definitions, biological activity, clinical use, etc.

DailyMed Regulated Products

The National Library of Medicine DailyMed is a searchable database that includes the most recent product labeling information submitted to the FDA by private companies.

NCATS GSRS Full Record

The Global Substance Registration System (GSRS) was designed to facilitate global monitoring of human and animal medicinal, food, tobacco, and cosmetic products. GSRS includes unique substance identifiers consistent with the ISO 11238 standard.