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Gene Therapy Trial Report

Summary

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
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

Therapy Information

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
Delivery System 🔄 Viral transduction
Vector Type 🔄 AAV9
Dose 1 🔄 1.1E14 vg/kg
Dose 2 🔄 1.5E14 vg/kg

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2022-11-02
Completion Date 2025-08
Last Update 2024-09-19

Participation Criteria

Eligible Age 1 Day - 180 Days
Standard Ages Child
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 2
Locations China

Regulatory Information

Has US IND False
FDA Designations
Recent Updates

Resources/Links

Clinical Publications 🔄
News and Press Releases 🔄
Preclinical Publications 🔄
Related NCTID 🔄