Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D94DTN
|
|||
| Drug Name |
BMN-307
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Phenylketonuria [ICD-11: 5C50.0; ICD-10: E70, E70.1] | Phase 1/2 | [1] | |
| Company |
BioMarin Pharmaceutical
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Phenylalanine hydroxylase (PAH) | Target Info | . | [1] |
| BioCyc | Phenylalanine degradation/tyrosine biosynthesis | |||
| KEGG Pathway | Phenylalanine metabolism | |||
| Phenylalanine, tyrosine and tryptophan biosynthesis | ||||
| Metabolic pathways | ||||
| Biosynthesis of amino acids | ||||
| Pathwhiz Pathway | Phenylalanine and Tyrosine Metabolism | |||
| WikiPathways | SIDS Susceptibility Pathways | |||
| Biogenic Amine Synthesis | ||||
| Metabolism of amino acids and derivatives | ||||
| Abnormal metabolism in phenylketonuria | ||||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04480567) A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects With Phenylketonuria. U.S.National Institutes of Health. | |||
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