Target Information
| Target General Information | Top | |||||
|---|---|---|---|---|---|---|
| Target ID |
T65941
|
|||||
| Target Name |
SERPINA1 messenger RNA (SERPINA1 mRNA)
|
|||||
| Synonyms |
Alpha-1 protease inhibitor; Alpha-1-antiproteinase; Serpin A1
Click to Show/Hide
|
|||||
| Gene Name |
SERPINA1
|
|||||
| Target Type |
Clinical trial target
|
[1] | ||||
| Disease | [+] 1 Target-related Diseases | + | ||||
| 1 | Alpha-1-antitrypsin deficiency [ICD-11: 5C5A] | |||||
| Function |
Inhibitor of serine proteases. Its primary target is elastase, but it also has a moderate affinity for plasmin and thrombin. Irreversibly inhibits trypsin, chymotrypsin and plasminogen activator. The aberrant form inhibits insulin-induced NO synthesis in platelets, decreases coagulation time and has proteolytic activity against insulin and plasmin.
Click to Show/Hide
|
|||||
| BioChemical Class |
mRNA target
|
|||||
| UniProt ID | ||||||
| Sequence |
MPSSVSWGILLLAGLCCLVPVSLAEDPQGDAAQKTDTSHHDQDHPTFNKITPNLAEFAFS
LYRQLAHQSNSTNIFFSPVSIATAFAMLSLGTKADTHDEILEGLNFNLTEIPEAQIHEGF QELLRTLNQPDSQLQLTTGNGLFLSEGLKLVDKFLEDVKKLYHSEAFTVNFGDTEEAKKQ INDYVEKGTQGKIVDLVKELDRDTVFALVNYIFFKGKWERPFEVKDTEEEDFHVDQVTTV KVPMMKRLGMFNIQHCKKLSSWVLLMKYLGNATAIFFLPDEGKLQHLENELTHDIITKFL ENEDRRSASLHLPKLSITGTYDLKSVLGQLGITKVFSNGADLSGVTEEAPLKLSKAVHKA VLTIDEKGTEAAGAMFLEAIPMSIPPEVKFNKPFVFLMIEQNTKSPLFMGKVVNPTQK Click to Show/Hide
|
|||||
| Drugs and Modes of Action | Top | |||||
|---|---|---|---|---|---|---|
| Clinical Trial Drug(s) | [+] 3 Clinical Trial Drugs | + | ||||
| 1 | Fazirsiran | Drug Info | Phase 3 | Alpha-1 antitrypsin deficiency | [2] | |
| 2 | ARO-AAT | Drug Info | Phase 2 | Alopecia | [3] | |
| 3 | Belcesiran | Drug Info | Phase 2 | Alpha-1 antitrypsin deficiency | [4] | |
| Cell-based Target Expression Variations | Top | |||||
|---|---|---|---|---|---|---|
| Cell-based Target Expression Variations | ||||||
| References | Top | |||||
|---|---|---|---|---|---|---|
| REF 1 | Clinical pipeline report, company report or official report of Arrowhead Pharmaceuticals. | |||||
| REF 2 | ClinicalTrials.gov (NCT05677971) A Randomized, Double-blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Fazirsiran in the Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease With METAVIR Stage F2 to F4 Fibrosis. U.S.National Institutes of Health. | |||||
| REF 3 | ClinicalTrials.gov (NCT03946449) Study of ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD). U.S. National Institutes of Health. | |||||
| REF 4 | ClinicalTrials.gov (NCT04764448) A Phase 2, Randomized, Double-blind, Placebo-Controlled Study Investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two Dose Levels of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency-Associated Liver Disease. U.S.National Institutes of Health. | |||||
| REF 5 | Fazirsiran for Liver Disease Associated with Alpha(1)-Antitrypsin Deficiency. N Engl J Med. 2022 Aug 11;387(6):514-524. | |||||
| REF 6 | Clinical pipeline report, company report or official report of Dicerna Pharmaceuticals | |||||
If You Find Any Error in Data or Bug in Web Service, Please Kindly Report It to Dr. Zhou and Dr. Zhang.