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Clinical Trial Report

Gene Therapy Trial Report

Summary

GCB-002 in Treatment of Patients with Rett Syndrome


NCTID NCT06739434 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Rett Syndrome
Disease Ontology Term DOID:1206
Compound Name GCB-002
Sponsor Genecombio Ltd.
Funder Type Other
Recruitment Status
Enrolling by invitation
Enrollment Count 6
Results Posted Not Available

Therapy Information


Target Gene/Variant MECP2
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intrathecal
Drug Product Type Viral vector
Target Tissue/Cell
Delivery System Viral transduction
Vector Type scAAV9
Editor Type
Dose 1 2 cohort dose escalation, undisclosed concentrations
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Na
Submit Date 2024-12-12
Completion Date 2030-12
Last Update 2025-03-11

Participation Criteria


Eligible Age 2 Years - 10 Years
Standard Ages Child
Sexes Eligible for Study FEMALE

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links


Resources/Links

No External Links Available.