Details of the Drug

General Information of Drug (ID: DM0N71R)

Drug Name

VY-AADC01

Indication

Disease Entry	ICD 11	Status	REF
Parkinson disease	8A00.0	Phase 1	[1]
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Drug Type

Gene therapy

Cross-matching ID

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	Not Available	[2]

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Molecular Interaction Atlas (MIA)

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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
Aromatic-L-amino-acid decarboxylase (DDC)	DTT	DDC	7.84E-03	-1.49	-1.01

Molecular Expression Atlas (MEA)

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References

1	ClinicalTrials.gov (NCT03065192) An Open-label Safety and Efficacy Study of VY-AADC01 Administered by MRI-Guided Convective Infusion Using a Posterior Trajectory Into the Putamen of Participants With Parkinson's Disease With Fluctuating Responses to Levodopa. U.S.National Institutes of Health.
2	Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes From the PD-1101 Trial. Neurology. 2022 Jan 4;98(1):e40-e50.