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Clinical Trial Report

Gene Therapy Trial Report

Summary

A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy.

NCTID NCT06392724 (View at clinicaltrials.gov)
Description
Indication Duchenne Muscular Dystrophy
Compound Name GEN6050X
Sponsor Peking Union Medical College Hospital
Funder Type Other
Status
Recruiting
Enrollment Count 3

Therapy Information

Target Gene/Variant DMD
Therapy Type Gene editing
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 eTAM
Dose 1 5 x 10^13 vg/kg body weight
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Early phase1
Submit Date 2024-04-26
Completion Date 2027-12
Last Update 2024-07-08

Participation Criteria

Eligible Age 4 Years - 10 Years
Standard Ages Child
Eligible Sex MALE

Locations

No.of Trial Sites 1
Locations China

Regulatory Information

Has US IND False
Recent Updates

Resources/Links

Patents
  • CA3191505A1
  • WO2017215619A1
Preclinical Publications