Safety and Pharmacodynamics of GNSC-001 Intra-articular Injection for Knee Osteoarthritis

NCTID	NCT05835895 (View at clinicaltrials.gov)
Description	The goal of this clinical study is to determine if a single injection of 1 or more dose levels of adeno-associated virus (AAV) gene therapy (GNSC-001) is safe and tolerated compared to placebo in participants with painful osteoarthritis (OA) of the knee. (Show More)
Development Status	Active
Indication	Osteoarthritis, Knee
Disease Ontology Term	DOID:8398
Compound Name	GNSC-001
Compound Description	AAV-CMV-IL1RN
Sponsor	Genascence Corporation
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	67
Results Posted	Not Available

Target Gene/Variant	IL1RN
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Overexpression of protective allele/gene
Route of Administration	Intraarticular
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2.5
Editor Type	none
Dose 1	1E11 vg/knee
Dose 2	1E12 vg/knee
Dose 3	1E13 vg/knee
Dose 4
Dose 5

No.of Trial Sites	9
Locations	United States

Has US IND	True
FDA Designations	Fast Track
Recent Updates	Initial data for Phase 1b trial expected Q4 2024