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Clinical Trial Report

Gene Therapy Trial Report

Summary

Safety and Efficacy of GS-100 Gene Therapy in Patients with NGLY1 Deficiency

NCTID NCT06199531 (View at clinicaltrials.gov)
Description
Development Status Active
Indication NGLY1 Deficiency
Disease Ontology Term DOID:0060728
Compound Name GS-100
Sponsor Grace Science, LLC
Funder Type Industry
Recruitment Status
Recruiting
Enrollment Count 6
Results Posted Not Available

Therapy Information

Target Gene/Variant NGLY1
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 none
Dose 1 Undisclosed low dose
Dose 2 Undisclosed medium dose
Dose 3 Undisclosed high dose
Dose 4
Dose 5

Study Record Dates

Current Stage Phase1, Phase2
Submit Date 2023-11-21
Completion Date 2028-01-31
Last Update 2025-03-25

Participation Criteria

Eligible Age 2 Years - 18 Years
Standard Ages Child, Adult
Sexes Eligible for Study ALL

Locations

No.of Trial Sites 3
Locations United States

Regulatory Information

Has US IND True
FDA Designations Support for Clinical Trials Advancing Rare Disease Therapeutics
Recent Updates Selected for START program (6/3/24)

Resources/Links

News and Press Releases
Preclinical Publications