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Clinical Trial Report

Gene Therapy Trial Report

Summary

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients


NCTID NCT05824169 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Spinal Muscular Atrophy
Disease Ontology Term DOID:13137
Compound Name GC101
Compound Description scAAV9.CBA.CMV.coSMN1
Sponsor GeneCradle Inc
Funder Type Industry
Recruitment Status
Recruiting
Enrollment Count 18
Results Posted Not Available

Therapy Information


Target Gene/Variant SMN1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intrathecal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV9
Editor Type
Dose 1 2.4E14 vg
Dose 2 4.8E14 vg
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2023-04-10
Completion Date 2025-12
Last Update 2023-04-25

Participation Criteria


Eligible Age 0 Months - 6 Months
Standard Ages Child
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 4
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links