A Study of PBFT02 in Patients With Frontotemporal Dementia and Progranulin Mutations (FTD-GRN)

NCTID	NCT04747431 (View at clinicaltrials.gov)
Description	PBFT02 is a gene therapy for frontotemporal dementia intended to deliver a functional copy of the GRN gene to the brain. This study will assess the safety, tolerability and efficacy of this treatment in patients with frontotemporal dementia and mutations in the progranulin gene (FTD-GRN). (Show More)
Indication	Frontotemporal Dementia
Compound Name	PBFT02
Sponsor	Passage Bio, Inc.
Funder Type	Industry
Status	Recruiting
Enrollment Count	15

Target Gene/Variant	GRN
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intracisterna magna
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV1
Editor Type
Dose 1	3.3 x 10^10 GC/g brain weight
Dose 2	1.1 x 10^11 GC/g brain weight
Dose 3	2.2 x 10^11 GC/g brain weight
Dose 4
Dose 5

No.of Trial Sites	8
Locations	Canada,United States,Brazil,Italy,Portugal

Has US IND	True
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