Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DG4X8E
|
|||
| Drug Name |
AT845
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Pompe disease [ICD-11: 5C51.3; ICD-10: E74.0] | Phase 2 | [1] | |
| Glycogen storage disease type II [ICD-11: 5C51.3] | Phase 1/2 | [1] | ||
| Company |
Audentes Therapeutics
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Lysosomal alpha-glucosidase (GAA) | Target Info | . | [2] |
| KEGG Pathway | Galactose metabolism | |||
| Starch and sucrose metabolism | ||||
| Metabolic pathways | ||||
| Lysosome | ||||
| Pathwhiz Pathway | Galactose Metabolism | |||
| Pathway Interaction Database | Notch-mediated HES/HEY network | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04174105) A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients With Late Onset Pompe Disease. U.S.National Institutes of Health. | |||
| REF 2 | Muscle-directed gene therapy corrects Pompe disease and uncovers species-specific GAA immunogenicity. EMBO Mol Med. 2022 Jan 11;14(1):e13968. | |||
If You Find Any Error in Data or Bug in Web Service, Please Kindly Report It to Dr. Zhou and Dr. Zhang.