Details of the Drug Therapeutic Target (DTT)

General Information of Drug Therapeutic Target (DTT) (ID: TTA7UJC)

• DTT Name: Alpha-1-antitrypsin (SERPINA1)
• Synonyms: SERPINA1; PRO0684/PRO2209; Alpha1-proteinase; Alpha-1-antiproteinase; Alpha-1 protease inhibitor
• Gene Name: SERPINA1
• DTT Type: Successful target; [1]
• BioChemical Class: Serpin protein
• UniProt ID: A1AT_HUMAN
• TTD ID: T28265
• Sequence:
  MPSSVSWGILLLAGLCCLVPVSLAEDPQGDAAQKTDTSHHDQDHPTFNKITPNLAEFAFS
  LYRQLAHQSNSTNIFFSPVSIATAFAMLSLGTKADTHDEILEGLNFNLTEIPEAQIHEGF
  QELLRTLNQPDSQLQLTTGNGLFLSEGLKLVDKFLEDVKKLYHSEAFTVNFGDTEEAKKQ
  INDYVEKGTQGKIVDLVKELDRDTVFALVNYIFFKGKWERPFEVKDTEEEDFHVDQVTTV
  KVPMMKRLGMFNIQHCKKLSSWVLLMKYLGNATAIFFLPDEGKLQHLENELTHDIITKFL
  ENEDRRSASLHLPKLSITGTYDLKSVLGQLGITKVFSNGADLSGVTEEAPLKLSKAVHKA
  VLTIDEKGTEAAGAMFLEAIPMSIPPEVKFNKPFVFLMIEQNTKSPLFMGKVVNPTQK
• Function: Inhibitor of serine proteases. Its primary target is elastase, but it also has a moderate affinity for plasmin and thrombin.
• KEGG Pathway: Complement and coagulation cascades (hsa04610)
• Reactome Pathway:
  COPII (Coat Protein 2) Mediated Vesicle Transport (R-HSA-204005)
  Cargo concentration in the ER (R-HSA-5694530)
  Platelet degranulation (R-HSA-114608)

Molecular Interaction Atlas (MIA) of This DTT

2 Approved Drug(s) Targeting This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Glassia	DM83MOK	Emphysema	CA21	Approved	[1]
Zemaira	DMA5QPM	Emphysema	CA21	Approved	[2]

2 Clinical Trial Drug(s) Targeting This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
AGTC-0106	DMMKASG	Alpha-1 antitrypsin deficiency	5C5A	Phase 2	[3]
Igmesine	DMH97XA	Major depressive disorder	6A70.3	Phase 2	[4]

1 Investigative Drug(s) Targeting This DTT

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
2-Sulfhydryl-Ethanol	DMJBO3D	Discovery agent	N.A.	Investigative	[5]

Molecular Expression Atlas (MEA) of This DTT

Disease Name	ICD 11	Studied Tissue	p-value	Fold-Change	Z-score
Chronic obstructive pulmonary disease	CA23	Lung tissue	7.84E-01	-0.08	-0.1
Chronic obstructive pulmonary disease	CA23	Small airway epithelium	1.32E-03	0.33	0.54

References

• [1] Mullard A: 2010 FDA drug approvals. Nat Rev Drug Discov. 2011 Feb;10(2):82-5.
• [2] Drugs@FDA. U.S. Food and Drug Administration. U.S. Department of Health Human Services.
• [3] First gene therapy nears landmark European market authorization. Nat Biotechnol. 2012 Sep;30(9):807-9.
• [4] Emerging drugs for the treatment of chronic obstructive pulmonary disease. Expert Opin Emerg Drugs. 2006 May;11(2):275-91.
• [5] DrugBank 3.0: a comprehensive resource for 'omics' research on drugs. Nucleic Acids Res. 2011 Jan;39(Database issue):D1035-41.