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Gene Therapy Trial Browser
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
Open-label, single dose clinical trial of scAAV9.U7.ACCA via peripheral limb vein injection for Duchenne muscular dystrophy boys who have a duplication of exon 2.
(Show More)
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
Clinical Publications
(Abstract) Expression of apparent full-length dystrophin in skeletal muscle in a first-in-human gene therapy trial using the scAAV9.U7-ACCA vector
News and Press Releases
Notice Regarding Impairment Loss for Products under Development
Preclinical Publications
(Abstract P13) Comparison of U7snRNA-induced dystrophin expression following systemic delivery with AAV9, MyoAAV 2A, and MyoAAV 3A capsids in the Dup2 mouse
Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping
Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping
Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model
[Webinar] Understanding Gene Therapy, Part 3 - Galgt2 and Dup2 - December 2017