An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy

NCTID	NCT06288230 (View at clinicaltrials.gov)
Description	This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients. (Show More)
Development Status	Active
Indication	Spinal Muscular Atrophy
Disease Ontology Term	DOID:13137
Compound Name	Vesemnogene lantuparvovec
Compound Description	AAV-hSMN1
Sponsor	Lantu Biopharma
Funder Type	Industry
Recruitment Status	Recruiting
Enrollment Count	6
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	AAV
Editor Type
Dose 1	Undisclosed dose
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China

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