Details of the Drug

General Information of Drug (ID: DMJ42E6)

Drug Name
ProSavin
Synonyms Dopamine synthesis pathway gene therapy (Parkinson's disease), Oxford BioMedica
Indication
Disease Entry ICD 11 Status REF
Parkinson disease 8A00.0 Phase 1/2 [1]
Cross-matching ID
TTD ID
D05ICS

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Aromatic-L-amino-acid decarboxylase (DDC) TTN451K DDC_HUMAN Modulator [2]
GTP cyclohydrolase-I (GCH1) TTLSWP6 GCH1_HUMAN Modulator [2]
Tyrosine 3-monooxygenase (TH) TTUHP71 TY3H_HUMAN Modulator [2]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

Molecular Expression Atlas of This Drug

The Studied Disease Parkinson disease
ICD Disease Classification 8A00.0
Molecule Name Molecule Type Gene Name p-value Fold-Change Z-score
Tyrosine 3-monooxygenase (TH) DTT TH 9.10E-04 -2.73 -1.55
Aromatic-L-amino-acid decarboxylase (DDC) DTT DDC 7.84E-03 -1.49 -1.01
GTP cyclohydrolase-I (GCH1) DTT GCH1 3.38E-02 -1.59 -1.53
Molecular Expression Atlas (MEA) Jump to Detail Molecular Expression Atlas of This Drug

References

1 ClinicalTrials.gov (NCT01856439) Long Term Safety and Efficacy Study of ProSavin in Parkinson's Disease. U.S. National Institutes of Health.
2 Clinical pipeline report, company report or official report of Oxford BioMedica.
3 GTP cyclohydrolase I feedback regulatory protein-dependent and -independent inhibitors of GTP cyclohydrolase I. Arch Biochem Biophys. 2001 Apr 1;388(1):67-73.
4 Clinical pipeline report, company report or official report of Oxford BioMedica.
5 The Protein Data Bank. Nucleic Acids Res. 2000 Jan 1;28(1):235-42.
6 How many drug targets are there Nat Rev Drug Discov. 2006 Dec;5(12):993-6.
7 Functional COMT variant predicts response to high dose pyridoxine in Parkinson's disease. Am J Med Genet B Neuropsychiatr Genet. 2005 Aug 5;137B(1):1-4.
8 Catechol-O-methyltransferase inhibitors in the management of Parkinson's disease. Semin Neurol. 2001;21(1):15-22.
9 The aromatic-L-amino acid decarboxylase inhibitor carbidopa is selectively cytotoxic to human pulmonary carcinoid and small cell lung carcinoma cells. Clin Cancer Res. 2000 Nov;6(11):4365-72.
10 UCSF report
11 Clinical pipeline report, company report or official report of Voyager Therapeutics.
12 Clinical pipeline report, company report or official report of PTC Therapeutics.
13 Activation of an adrenergic pro-drug through sequential stereoselective action of tandem target enzymes. Biochem Biophys Res Commun. 1992 Nov 30;189(1):33-9.
14 URL: http://www.guidetopharmacology.org Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Target id: 1271).
15 Selectivity and affinity determinants for ligand binding to the aromatic amino acid hydroxylases. Curr Med Chem. 2007;14(4):455-67.
16 Effect of metals and phenylalanine on the activity of human tryptophan hydroxylase-2: comparison with that on tyrosine hydroxylase activity. Neurosci Lett. 2006 Jul 3;401(3):261-5.
17 Biochemistry of postmortem brains in Parkinson's disease: historical overview and future prospects. J Neural Transm Suppl. 2007;(72):113-20.
18 Dopamine beta-hydroxylase deficiency. A genetic disorder of cardiovascular regulation. Hypertension. 1991 Jul;18(1):1-8.
19 Effect of L1-79 on Core Symptoms of Autism Spectrum Disorder: A Case Series. Clin Ther. 2019 Oct;41(10):1972-1981.
20 URL: http://www.guidetopharmacology.org Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Target id: 1243).