Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

NCTID	NCT02716246 (View at clinicaltrials.gov)
Description	The main objective of this study is to evaluate the efficacy and safety of ABO-102 for the treatment of MPS IIIA. (Show More)
Indication	Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Compound Name	Rebisufligene etisparvovec (scAAV9.U1a.hSGSH)
Sponsor	Ultragenyx Pharmaceutical Inc
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	28

Target Gene/Variant	SGSH
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV9
Editor Type
Dose 1	0.5 x 10^13 vg/kg
Dose 2	1.0 x 10^13 vg/kg
Dose 3	3.0 x 10^13 vg/kg
Dose 4
Dose 5

No.of Trial Sites	5
Locations	United States,Australia,Spain

Has US IND	True
Recent Updates	Accelerated Approval BLA filing planned for 2024/early 2025