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Clinical Trial Report

Gene Therapy Trial Report

Summary

AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.


NCTID NCT04240314 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Duchenne Muscular Dystrophy (DMD)
Disease Ontology Term DOID:11723
Compound Name AT702
Compound Description scAAV9.U7.ACCA
Sponsor Megan Waldrop
Funder Type Other
Recruitment Status
Active not recruiting
Enrollment Count 3
Results Posted Not Available

Therapy Information


Target Gene/Variant DMD (exon 2)
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Exon skipping/splice editor
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV9
Editor Type
Dose 1 3E13 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2020-01-22
Completion Date 2025-11-19
Last Update 2023-02-09

Participation Criteria


Eligible Age 6 Months - 13 Years
Standard Ages Child
Sexes Eligible for Study MALE

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Product was licensed to Audentes/Astellas Therapeutics in 2019, discontinuation was announced in April 2022

Resources/Links