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Gene Therapy Trial Report

Summary

A Phase Ⅰ/Ⅱa Study of SNUG01 in Adult Subjects With ALS

NCTID NCT07169175 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Amyotrophic Lateral Sclerosis (ALS)
Disease Ontology Term DOID:332
Compound Name SNUG01
Sponsor SineuGene Therapeutics Co., Ltd.
Funder Type Industry
Recruitment Status
Not yet recruiting
Enrollment Count 21 (ESTIMATED)
Results Posted Not Available

Therapy Information

Target Gene/Variant TRIM72
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Genetic delivery of therapeutic protein
Route of Administration Intrathecal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 3 undisclosed dose levels
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2025-08-08
Completion Date 2028-09-30
Last Update 2025-09-11

Participation Criteria

Eligible Age 18 Years - 80 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 4
Locations United States,China

Regulatory Information

Has US IND True
FDA Designations Orphan Drug Designation
Recent Updates FDA cleared IND application

Resources/Links

News and Press Releases
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