GDNF Gene Therapy for Multiple System Atrophy

NCTID	NCT04680065 (View at clinicaltrials.gov)
Description	The objective of this randomized, double-blinded, placebo-controlled Phase 1 investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a possible or probable diagnosis of Multiple System Atrophy. (Show More)
Indication	Multiple System Atrophy, Parkinsonian Subtype
Compound Name	AB-1005 (AAV2-GDNF)
Sponsor	Brain Neurotherapy Bio, Inc.
Funder Type	Industry
Status	Recruiting
Enrollment Count	9

Target Gene/Variant	GDNF
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intraparenchymal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2
Editor Type
Dose 1	Undisclosed dose 1
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	6
Locations	United States

Has US IND	True
Recent Updates	First patient randomized 11/17/23