VY-AADC02 for Parkinson's Disease With Motor Fluctuations (RESTORE-1)

NCTID	NCT03562494 (View at clinicaltrials.gov)
Description	The objective of this study is to assess the safety of VY-AADC02 in participants with Parkinson's disease (PD) with motor fluctuations. (Show More)
Development Status	Inactive
Indication	Parkinson's Disease
Disease Ontology Term	DOID:14330
Compound Name	VY-AADC01
Compound Description	AAV-hAADC-2
Sponsor	Neurocrine Biosciences
Funder Type	Industry
Recruitment Status	Completed
Enrollment Count	14
Results Posted	Not Available

Target Gene/Variant	DDC
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intraparenchymal (striatum)
Drug Product Type	Viral vector
Target Tissue/Cell	Striatum
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type
Dose 1	Total dose: 9E10 vg/200ul (4 sites)
Dose 2	Total dose: 3E11 vg/200ul (4 sites)
Dose 3	3.6E12 vg
Dose 4
Dose 5

No.of Trial Sites	11
Locations	United States

Has US IND	True
FDA Designations	Regenerative Medicine Advanced Therapy
Recent Updates	Avigen transferred AAV-based product rights to Genzyme Corporation in December 2005, Genzyme launched a collaboration with Voyager Therapeutics in February 2015, then this collaboration was dissolved by Sanofi-Genzyme in October 2017, Voyager entered a collaborative agreement with Neurocrine Biosciences Inc. in March 2019, Neurocrine terminated this agreement effective August 2021, Voyager announced they were terminating this program in 2022