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Clinical Trial Report

Gene Therapy Trial Report

Summary

An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

NCTID NCT06272149 (View at clinicaltrials.gov)
Description
Indication Gaucher Disease, Type 2
Compound Name VGN-R08b
Sponsor Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Funder Type Other
Status
Recruiting
Enrollment Count 6

Therapy Information

Target Gene/Variant GBA1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intracerebroventricular
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type AAV9
Editor Type
Dose 1 Undisclosed low dose
Dose 2 Undisclosed high dose
Dose 3
Dose 4
Dose 5

Study Record Dates

Current Stage Early phase1
Submit Date 2023-07-17
Completion Date 2029-02-28
Last Update 2024-02-22

Participation Criteria

Eligible Age 0 Months - 24 Months
Standard Ages Child
Eligible Sex ALL

Locations

No.of Trial Sites 1
Locations China

Regulatory Information

Has US IND False
Recent Updates FIH interim data presented at ASGCT 2024

Resources/Links

Patents
  • WO2024017387A1
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