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Clinical Trial Report

Gene Therapy Trial Report

Summary

A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients with Type I Gaucher Disease


NCTID NCT06818838 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Gaucher Disease Type 1
Disease Ontology Term DOID:0110957
Compound Name LY-M001
Sponsor Lingyi Biotech Co., Ltd.
Funder Type Industry
Recruitment Status
Recruiting
Enrollment Count 18
Results Posted Not Available

Therapy Information


Target Gene/Variant GBA1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell Liver
Delivery System Viral transduction
Vector Type AAV8
Editor Type
Dose 1 Starting dose: 1.5E13 vg/kg
Dose 2 High dose: 3.0E13 vg/kg
Dose 3 Backdose: 5E12 vg/kg
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2025-01-24
Completion Date 2031-07-30
Last Update 2025-02-13

Participation Criteria


Eligible Age 18 Years - 60 Years
Standard Ages Adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates IND granted in January 2024

Resources/Links


Resources/Links

No External Links Available.