Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

NCTID	NCT04713475 (View at clinicaltrials.gov)
Description	PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues. This study will assess in a 2 part design the safety, tolerability and efficacy of PBGM01 in patients with early onset infantile (Type 1) and late onset infantile (Type 2a) GM1 gangliosidosis (Show More)
Indication	GM1 Gangliosidosis
Compound Name	PBGM01
Sponsor	Passage Bio, Inc.
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	26

Target Gene/Variant	GLB1
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	AAVhu68
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	TBD
Dose 5

No.of Trial Sites	9
Locations	Canada,Turkey,United States,Brazil,United Kingdom

Has US IND	True
Recent Updates	Out-licensed development to Gemma Biotherapeutics, uncertain development status