Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1

NCTID	NCT05614531 (View at clinicaltrials.gov)
Description	The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307 as a treatment of spinal muscular atrophy Type 1 (SMN1). (Show More)
Development Status	Active
Indication	Spinal Muscular Atrophy Type I
Disease Ontology Term	DOID:13137
Compound Name	EXG001-307
Compound Description	scAAV9.CMVenh.hSyn.hSMN
Sponsor	Hangzhou Jiayin Biotech Ltd
Funder Type	Industry
Recruitment Status	Enrolling by invitation
Enrollment Count	12 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	SMN1
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	1.1E14 vg/kg
Dose 2	1.5E14 vg/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	China