Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D47RIS
|
|||
| Drug Name |
OTL-101
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Severe combined immunodeficiency [ICD-11: 4A01.10] | Phase 3 | [1] | |
| Company |
Orchard Therapeutics
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Adenosine deaminase (ADA) | Target Info | Replacement | [2] |
| BioCyc | Purine nucleotides degradation | |||
| Purine deoxyribonucleosides degradation | ||||
| Purine ribonucleosides degradation to ribose-1-phosphate | ||||
| Adenosine nucleotides degradation | ||||
| Superpathway of purine nucleotide salvage | ||||
| Adenine and adenosine salvage III | ||||
| KEGG Pathway | Purine metabolism | |||
| Metabolic pathways | ||||
| Primary immunodeficiency | ||||
| NetPath Pathway | TCR Signaling Pathway | |||
| IL2 Signaling Pathway | ||||
| Panther Pathway | Adenine and hypoxanthine salvage pathway | |||
| Pathwhiz Pathway | Purine Metabolism | |||
| Pathway Interaction Database | p73 transcription factor network | |||
| C-MYB transcription factor network | ||||
| Validated transcriptional targets of deltaNp63 isoforms | ||||
| Validated transcriptional targets of TAp63 isoforms | ||||
| Reactome | Purine salvage | |||
| WikiPathways | Metabolism of nucleotides | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04140539) A Clinical Study to Enable Process Validation of Commercial Grade OTL-101. U.S. National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of Orchard Therapeutics. | |||
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